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Genomes and Genes | David A LomasSummaryAffiliation: University of Cambridge Country: UK Publications
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Publications
Polymerisation underlies alpha1-antitrypsin deficiency, dementia and other serpinopathiesDavid A Lomas
Department of Medicine, University of Cambridge, Cambridge Institute for Medical Research, Wellcome Trust MRC Building, Hills Road, Cambridge CB2 2XY, United Kingdom
Front Biosci 9:2873-91. 2004..We have grouped these conditions together as the serpinopathies as recognition of their common pathophysiology provides a platform to develop strategies to treat the associated clinical syndromes...
Hypersensitive mousetraps, alpha1-antitrypsin deficiency and dementiaD A Lomas
Respiratory Medicine Unit, Department of Medicine, University of Cambridge, Cambridge Institute for Medical Research, Wellcome Trust MRC Building, Hills Road, Cambridge CB2 2XY, U K
Biochem Soc Trans 30:89-92. 2002..This must now be achieved in vivo if we are to treat the associated clinical syndromes...
Structural dynamics associated with intermediate formation in an archetypal conformational diseaseMun Peak Nyon
Institute of Structural and Molecular Biology, Department of Biological Sciences, Birkbeck College, London, WC1E 7HX, UK
Structure 20:504-12. 2012..Conformational disease intermediates may best be defined using powerful but minimally perturbing techniques, mild disease mutants, and physiological conditions...
Stem cell-based therapy for α₁-antitrypsin deficiencyS Tamir Rashid
Department of Medicine, University of Cambridge, Cambridge Institute for Medical Research, Wellcome Trust MRC Building, Hills Road, Cambridge CB2 0XY, UK
Stem Cell Res Ther 3:4. 2012..Several important safety concerns will need to be addressed before this can be translated into clinical practice...
An oral inhibitor of p38 MAP kinase reduces plasma fibrinogen in patients with chronic obstructive pulmonary diseaseDavid A Lomas
Department of Medicine, University of Cambridge, Cambridge Institute for Medical Research, Wellcome Trust MRC Building, Hills Road, Cambridge, CB2 0XY, UK
J Clin Pharmacol 52:416-24. 2012..87; 95% confidence interval, 0.82-0.93; P < .001). It was concluded that oral losmapimod significantly reduced plasma fibrinogen in patients with COPD...
Characterisation of COPD heterogeneity in the ECLIPSE cohortAlvar Agusti
Thorax Institute, Hospital Clinic, IDIBAPS, Universitat de Barcelona, Spain
Respir Res 11:122. 2010..Chronic obstructive pulmonary disease (COPD) is a complex condition with pulmonary and extra-pulmonary manifestations. This study describes the heterogeneity of COPD in a large and well characterised and controlled COPD cohort (ECLIPSE)...
Association between neuroserpin and molecular markers of brain damage in patients with acute ischemic strokeRaquel Rodríguez-González
Clinical Neuroscience Research Laboratory, Department of Neurology, Hospital Clinico Universitario, University of Santiago de Compostela, Santiago de Compostela, Spain
J Transl Med 9:58. 2011..Our aim was to study whether neuroserpin serum levels could be associated to biomarkers of excitotoxicity, inflammation and blood brain barrier disruption...
Evaluation of serum CC-16 as a biomarker for COPD in the ECLIPSE cohortD A Lomas
Department of Medicine, University of Cambridge, Cambridge Institute for Medical Research, Wellcome Trust MRC Building, Hills Road, Cambridge CB2 0XY, UK
Thorax 63:1058-63. 2008..Circulating levels of Clara cell secretory protein-16 (CC-16) have been linked to Clara cell toxicity. It has therefore been suggested that this protein may be a useful marker of chronic obstructive pulmonary disease (COPD)...
Parker B. Francis lectureship. Antitrypsin deficiency, the serpinopathies, and chronic obstructive pulmonary diseaseDavid A Lomas
Department of Medicine, Cambridge Institute for Medical Research, Wellcome Trust MRC Building, University of Cambridge, Hills Road, Cambridge CB2 2XY, UK
Proc Am Thorac Soc 3:499-501. 2006..This conformational transition may explain the excessive inflammation that underlies the progressive emphysema associated with Z alpha(1)-antitrypsin deficiency...
Serpinopathies and the conformational dementiasDavid A Lomas
Department of Medicine, University of Cambridge, Cambridge Institute for Medical Research, Wellcome Trust MRC Building, Hills Road, Cambridge CB2 2XY, UK
Nat Rev Genet 3:759-68. 2002....
Alpha1-antitrypsin polymerization and the serpinopathies: pathobiology and prospects for therapyDavid A Lomas
Respiratory Medicine Unit, Department of Medicine, University of Cambridge, Cambridge Institute for Medical Research, Cambridge, United Kingdom
J Clin Invest 110:1585-90. 2002
The selective advantage of alpha1-antitrypsin deficiencyDavid A Lomas
Department of Medicine, University of Cambridge, Cambridge Institute for Medical Research, Wellcome Trust MRC Building, Hills Road, Cambridge, CB2 2XY UK
Am J Respir Crit Care Med 173:1072-7. 2006....
The genetics of chronic obstructive pulmonary diseaseD A Lomas
Department of Medicine, University of Cambridge, Cambridge, UK
Respir Res 2:20-6. 2001..Many candidate genes have been assessed, but the data are often conflicting. We review the genetic factors that predispose smokers to COPD and highlight the future role of genomic scans in identifying novel susceptibility genes...
Molecular mousetraps, alpha1-antitrypsin deficiency and the serpinopathiesDavid A Lomas
Department of Medicine, University of Cambridge, Cambridge Institute for Medical Research
Clin Med 5:249-57. 2005..In view of the common mechanism underlying these conditions, we have grouped them together as the serpinopathies...
Alpha1-antitrypsin deficiency. 4: Molecular pathophysiologyD A Lomas
Respiratory Medicine Unit, Department of Medicine, University of Cambridge, Cambridge Institute for Medical Research, Cambridge CB2 2XY, UK
Thorax 59:529-35. 2004..The current goals are to determine the cellular response to polymeric alpha(1)-antitrypsin and to develop therapeutic strategies to block polymerisation in vivo...
Loop-sheet polymerization: the mechanism of alpha1-antitrypsin deficiencyD A Lomas
Respiratory Medicine Unit, Department of Medicine, University of Cambridge, Wellcome Trust Centre for Molecular Mechanisms in Disease, Cambridge Institute for Medical Research, UK
Respir Med 94:S3-6. 2000..Understanding this mechanism of polymerization allows rational drug design to block the protein-protein linkage and so ameliorate the associated disease...
Serum surfactant protein D is steroid sensitive and associated with exacerbations of COPDD A Lomas
Department of Medicine, University of Cambridge, Cambridge Institute for Medical Research, Wellcome Trust MRC Building, Hills Road, Cambridge, CB2 0XY, UK
Eur Respir J 34:95-102. 2009..It may represent an intermediate measure for the development of novel anti-inflammatory agents...
Molecular mousetraps and the serpinopathiesD A Lomas
Department of Medicine, University of Cambridge, Cambridge Institute for Medical Research, Wellcome Trust MRC Building, Hills Road, Cambridge CB2 2XY, UK
Biochem Soc Trans 33:321-30. 2005..We review in this paper the molecular and structural basis of the serpinopathies and show how this has allowed the development of specific agents to block the polymerization that underlies disease...
Genetic predisposition to chronic obstructive pulmonary disease: advances in alpha1-antitrypsin deficiency and the serpinopathiesDavid A Lomas
University of Cambridge, Cambridge Institute for Medical Research
Clin Med 7:446-7. 2007
Chronic obstructive pulmonary disease. IntroductionD A Lomas
Department of Medicine, University of Cambridge, Cambridge Institute for Medical Research, UK
Thorax 57:735. 2002....
The serpinopathies studying serpin polymerization in vivoJames A Irving
Department of Medicine, Cambridge Institute for Medical Research, University of Cambridge, Cambridge, United Kingdom
Methods Enzymol 501:421-66. 2011..We describe here the biochemical techniques, monoclonal antibodies, cell biology, animal models, and stem cell technology that are useful to characterize the serpin polymers that form in vivo...
The intracellular accumulation of polymeric neuroserpin explains the severity of the dementia FENIBElena Miranda
Department of Medicine, University of Cambridge, Cambridge Institute for Medical Research, Wellcome Trust MRC Building, Hills Road, Cambridge CB2 0XY, UK
Hum Mol Genet 17:1527-39. 2008..Moreover, mutant neuroserpin causes locomotor deficits in the fly allowing us to demonstrate a direct link between polymer accumulation and neuronal toxicity...
Endoplasmic reticulum-associated degradation (ERAD) and autophagy cooperate to degrade polymerogenic mutant serpinsHeike Kroeger
Department of Medicine, Cambridge Institute for Medical Research, Wellcome Trust MRC Building, University of Cambridge, Cambridge CB2 0XY, United Kingdom
J Biol Chem 284:22793-802. 2009..The significance of these findings to the treatment of serpinopathies is discussed...
Latent S49P neuroserpin forms polymers in the dementia familial encephalopathy with neuroserpin inclusion bodiesMaki Onda
Department of Medicine, University of Cambridge, Cambridge Institute for Medical Research, Wellcome Trust MRC Building, Hills Road, Cambridge CB2 2XY, United Kingdom
J Biol Chem 280:13735-41. 2005..These data provide an alternative method for the inactivation of mutant neuroserpin as a proteinase inhibitor in FENIB and demonstrate a second pathway for the formation of intracellular polymers in association with disease...
Sugar and alcohol molecules provide a therapeutic strategy for the serpinopathies that cause dementia and cirrhosisLynda K Sharp
Department of Medicine, University of Cambridge, UK
FEBS J 273:2540-52. 2006..These data demonstrate that sugar and alcohol molecules can reduce the polymerization of serpin mutants that cause disease, possibly by binding to and stabilizing beta-sheet A...
Iron promotes the toxicity of amyloid beta peptide by impeding its ordered aggregationBeinan Liu
Department of Medicine, Cambridge Institute for Medical Research, University of Cambridge, Wellcome Trust Medical Research Council Building, Hills Road, Cambridge CB2 0XY, United Kingdom
J Biol Chem 286:4248-56. 2011..These data support the hypothesis that iron delays the formation of well ordered aggregates of Aβ and so promotes its toxicity in Alzheimer disease...
Neuroserpin binds Abeta and is a neuroprotective component of amyloid plaques in Alzheimer diseaseKerri J Kinghorn
Department of Medicine, University of Cambridge, Cambridge Institute for Medical Research, Wellcome Trust Medical Research Council Building, Hills Road, Cambridge CB2 2XY, United Kingdom
J Biol Chem 281:29268-77. 2006..Taken together, these data show that neuroserpin interacts with Abeta(1-42) to form off-pathway non-toxic oligomers and so protects neurons in Alzheimer disease...
Neuroserpin Portland (Ser52Arg) is trapped as an inactive intermediate that rapidly forms polymers: implications for the epilepsy seen in the dementia FENIBDidier Belorgey
Cambridge Institute for Medical Research, Department of Medicine, University of Cambridge, UK
Eur J Biochem 271:3360-7. 2004..Moreover the inactivity of the mutant may result in uncontrolled activity of tissue plasminogen activator, and so explain the epileptic seizures seen in individuals with more severe forms of the disease...
Targeted metabolomics identifies perturbations in amino acid metabolism that sub-classify patients with COPDBaljit K Ubhi
Department of Biochemistry and the Cambridge Systems Biology Centre, University of Cambridge, UK
Mol Biosyst 8:3125-33. 2012..New methods were developed and validated to evaluate small molecules as potential diagnostic tools in patients with COPD, COPD related cachexia and cancer related cachexia...
Targeting a surface cavity of alpha 1-antitrypsin to prevent conformational diseaseHelen Parfrey
Respiratory Medicine Unit, Department of Medicine, University of Cambridge, Cambridge Institute for Medical Research, Wellcome Trust MRC Building, Hills Road, Cambridge CB2 2XY, United Kingdom
J Biol Chem 278:33060-6. 2003..These results demonstrate the importance of this cavity as a site for drug design to ameliorate polymerization and prevent the associated conformational disease...
Small molecules block the polymerization of Z alpha1-antitrypsin and increase the clearance of intracellular aggregatesMeera Mallya
Department of Medicine, University of Cambridge, Cambridge Institute for Medical Research, Wellcome Trust MRC Building, Cambridge CB2 2XY, UK
J Med Chem 50:5357-63. 2007..Identifying small molecules provides a novel therapy for the treatment of liver disease associated with the Z allele of alpha1-antitrypsin...
A novel monoclonal antibody to characterize pathogenic polymers in liver disease associated with alpha1-antitrypsin deficiencyElena Miranda
Department of Medicine, University of Cambridge, Cambridge Institute for Medical Research, Wellcome Trust Medical Research Council Building, Cambridge, UK
Hepatology 52:1078-88. 2010..CONCLUSION: Z and shutter domain mutants of alpha(1)-antitrypsin form polymers with a shared epitope and so are likely to have a similar structure...
Expression in drosophila of tandem amyloid β peptides provides insights into links between aggregation and neurotoxicityElena Speretta
Department of Genetics, University of Cambridge, Downing Street, Cambridge CB2 3EH, United Kingdom
J Biol Chem 287:20748-54. 2012....
Inhibitory activity of the Drosophila melanogaster serpin Necrotic is dependent on lysine residues in the D-helixAndrew S Robertson
Department of Genetics, University of Cambridge, Cambridge CB2 3EH, United Kingdom
J Biol Chem 281:26437-43. 2006..These data demonstrate a critical role for basic residues within the D-helix (and lysine 68 in particular) in the inhibitory mechanism of the serpin Necrotic...
alpha1-Antitrypsin deficiency, chronic obstructive pulmonary disease and the serpinopathiesUgo I Ekeowa
Department of Medicine, University of Cambridge, Cambridge Institute for Medical Research, Wellcome Trust MRC Building, Cambridge CB2 0XY, U K
Clin Sci (Lond) 116:837-50. 2009....
Neuroserpin polymers activate NF-kappaB by a calcium signaling pathway that is independent of the unfolded protein responseMark J Davies
Department of Medicine, University of Cambridge, Cambridge Institute for Medical Research, Wellcome Trust Medical Research Council Building, Hills Road, Cambridge CB2 0XY, United Kingdom
J Biol Chem 284:18202-9. 2009..Our results provide strong support for the endoplasmic reticulum overload response being independent of the unfolded protein response...
Characterisation of serpin polymers in vitro and in vivoDidier Belorgey
Dept of Medicine, University of Cambridge, Cambridge Institute for Medical Research, Wellcome Trust MRC Building, Hills Road, Cambridge CB2 0XY, UK
Methods 53:255-66. 2011..The characterisation of the pathological polymers requires heating of the folded protein or alternatively the assessment of ordered polymers from cell and animal models of disease or from the tissues of humans who carry the mutation...
Mutants of neuroserpin that cause dementia accumulate as polymers within the endoplasmic reticulumElena Miranda
Departments of Medicine and Clinical Biochemistry, University of Cambridge, Cambridge Institute for Medical Research, Wellcome Trust Medical Research Council Building, Hills Road, Cambridge CB2 2XY, United Kingdom
J Biol Chem 279:28283-91. 2004....
Mutant Neuroserpin (S49P) that causes familial encephalopathy with neuroserpin inclusion bodies is a poor proteinase inhibitor and readily forms polymers in vitroDidier Belorgey
Respiratory Medicine Unit and Neurology Unit, Department of Medicine, University of Cambridge, Cambridge Institute for Medical Research, Wellcome Trust Medical Research Council Building, United Kingdom
J Biol Chem 277:17367-73. 2002..These findings provide strong support for the role of neuroserpin polymerization in the formation of the intraneuronal inclusions that are characteristic of FENIB...
Probing neuroserpin polymerization and interaction with amyloid-beta peptides using single molecule fluorescenceAlbert Chiou
Department of Chemistry, University of Cambridge, Cambridge, United Kingdom
Biophys J 97:2306-15. 2009..The A beta(1-40) is displaced from the complex so that it acts as a catalyst and is not incorporated into neuroserpin polymers...
Sequestration of the Abeta peptide prevents toxicity and promotes degradation in vivoLeila M Luheshi
Department of Chemistry, University of Cambridge, Cambridge, United Kingdom
PLoS Biol 8:e1000334. 2010....
Defining the mechanism of polymerization in the serpinopathiesUgo I Ekeowa
Department of Medicine, University of Cambridge, Cambridge Institute for Medical Research, Wellcome Trust Medical Research Council Building, Cambridge CB2 0XY, United Kingdom
Proc Natl Acad Sci U S A 107:17146-51. 2010..Understanding the structure of the serpin polymer is essential for rational drug design strategies that aim to block polymerization and so treat α(1)-antitrypsin deficiency and the serpinopathies...
Practical genetics: alpha-1-antitrypsin deficiency and the serpinopathiesDamian C Crowther
1Department of Medicine, University of Cambridge, Cambridge Institute for Medical Research, Wellcome Trust/MRC Building, Hills Road, Cambridge CB2 2XY, UK
Eur J Hum Genet 12:167-72. 2004..We review here the genetic and molecular basis and clinical features of alpha(1)-antitrypsin deficiency, and show how this provides a platform to understand the other serpinopathies...
Inhibiting polymerization: new therapeutic strategies for Z alpha1-antitrypsin-related emphysemaHelen Parfrey
Division of Respiratory Medicine, Department of Medicine, University of Cambridge, Cambridge Institute for Medical Research, MRC Wellcome Trust Building, Hills Road, Cambridge CB2 2XY, UK
Am J Respir Cell Mol Biol 31:133-9. 2004..Thus, it raises the novel prospect of ameliorating both the cirrhosis and the emphysema associated with Z-AT...
COPD association and repeatability of blood biomarkers in the ECLIPSE cohortJennifer A Dickens
Department of Medicine, University of Cambridge, Cambridge Institute for Medical Research, Cambridge, UK
Respir Res 12:146. 2011..A panel of putative blood biomarkers was assessed in a subgroup of the Evaluation of COPD Longitudinally to Identify Surrogate Endpoints (ECLIPSE) cohort...
Detection of early locomotor abnormalities in a Drosophila model of Alzheimer's diseaseThomas R Jahn
Department of Genetics, University of Cambridge, UK Department of Chemistry, University of Cambridge, UK
J Neurosci Methods 197:186-9. 2011..The approach can be widely applied to different disease models in a number of model organisms...
6-mer peptide selectively anneals to a pathogenic serpin conformation and blocks polymerization. Implications for the prevention of Z alpha(1)-antitrypsin-related cirrhosisRavi Mahadeva
Respiratory Medicine Unit, Department of Medicine, University of Cambridge, Cambridge CB2 2XY, United Kingdom
J Biol Chem 277:6771-4. 2002..Furthermore they demonstrate how a conformational disease process can be selectively inhibited with a small peptide...
Intracellular serpins, firewalls and tissue necrosisStefan J Marciniak
Department of Medicine, University of Cambridge, Cambridge Institute for Medical Research, Wellcome Trust MRC Building, Hills Road, Cambridge CB2 0XY, UK
Trends Cell Biol 18:45-7. 2008..We discuss how mutant serpins cause disease either through polymerization or now, perhaps, by unleashing necrosis...
Alpha1-antitrypsin deficiency--a model for conformational diseasesRobin W Carrell
Department of Hematology, University of Cambridge, Cambridge Institute for Medical Research, Cambridge, United Kingdom
N Engl J Med 346:45-53. 2002
pH-dependent stability of neuroserpin is mediated by histidines 119 and 138; implications for the control of beta-sheet A and polymerizationDidier Belorgey
Department of Medicine, University of Cambridge, Cambridge Institute for Medical Research, Cambridge CB2 0XY, United Kingdom
Protein Sci 19:220-8. 2010..This mechanism is likely to have evolved to protect neuroserpin from the acidic environment of the secretory granules...
Nucleation of alpha 1-antichymotrypsin polymerizationDamian C Crowther
Neurology Unit, University of Cambridge, Cambridge Institute for Medical Research, Wellcome Trust MRC Building, Hills Road, Cambridge, CB2 2XY, United Kingdom
Biochemistry 42:2355-63. 2003..This mechanism is analogous to the fibrillization of the Abeta(1-42) peptide and may be important in the deposition of alpha(1)-antichymotrypsin in the plaques of Alzheimer's disease...
Protein misfolding and the serpinopathiesDidier Belorgey
Department of Medicine, University of Cambridge, Cambridge Institute for Medical Research, Cambridge, United Kingdom
Prion 1:15-20. 2007..The common mechanism of polymerization has allowed us to group these conditions together as a novel class of disease, the serpinopathies...
Characterization of the necrotic protein that regulates the Toll-mediated immune response in DrosophilaAndrew S Robertson
Department of Genetics, University of Cambridge, Downing Street, Cambridge CB2 3EH, United Kingdom
J Biol Chem 278:6175-80. 2003..These data show that Necrotic is likely to inhibit a wide range of proteases in Drosophila and that Nec has the specificity requirements to act as the physiological inhibitor of Persephone in vivo...
Intrinsic determinants of neurotoxic aggregate formation by the amyloid beta peptideAnn Christin Brorsson
Department of Chemistry, University of Cambridge, Cambridge, United Kingdom
Biophys J 98:1677-84. 2010....
Endoplasmic reticulum dysfunction in neurological diseaseBenoit D Roussel
Department of Medicine, Cambridge Institute for Medical Research, University of Cambridge, Cambridge, UK
Lancet Neurol 12:105-18. 2013..By targeting components of these signalling responses, amelioration of their toxic effects and so the treatment of a range of neurodegenerative disorders might become possible...
Polymers of Z alpha1-antitrypsin co-localize with neutrophils in emphysematous alveoli and are chemotactic in vivoRavi Mahadeva
Department of Medicine, Box 157, Level 5, Addenbrookes NHS Trust, Hills Road, Cambridge CB2 2QQ, UK
Am J Pathol 166:377-86. 2005..These findings may also explain the progression of emphysema in some individuals despite alpha(1)-antitrypsin replacement therapy...
The iFly tracking system for an automated locomotor and behavioural analysis of Drosophila melanogasterKai J Kohlhoff
Department of Chemistry, University of Cambridge, Cambridge, UK
Integr Biol (Camb) 3:755-60. 2011....
Impaired tissue growth is mediated by checkpoint kinase 1 (CHK1) in the integrated stress responseElke Malzer
Department of Medicine, University of Cambridge, Cambridge Institute for Medical Research CIMR, Wellcome Trust MRC Building, Hills Road, Cambridge, CB2 0XY, UK
J Cell Sci 123:2892-900. 2010..PERK was both necessary and sufficient for CHK1 activation. These findings indicate that non-genotoxic misfolded protein stress accesses DNA-damage-induced cell cycle checkpoints to couple the ISR to cell cycle arrest...
Unravelling the twists and turns of the serpinopathiesBenoit D Roussel
Department of Medicine, University of Cambridge, Cambridge Institute for Medical Research, Wellcome Trust MRC Building, Cambridge, UK
FEBS J 278:3859-67. 2011..These include the use of small molecules to block polymerization, stimulation of autophagy to clear inclusions and stem cell technology to correct the underlying molecular defect...
p53 and translation attenuation regulate distinct cell cycle checkpoints during endoplasmic reticulum (ER) stressSally E Thomas
Department of Medicine and Cambridge Institute for Medical Research, University of Cambridge, Wellcome Trust Medical Research Council Building, Hills Road, Cambridge CB2 0XY, United Kingdom
J Biol Chem 288:7606-17. 2013..We propose that cell cycle regulation in response to ER stress comprises redundant pathways invoked sequentially first to impair G(2) progression prior to ultimate G(1) arrest...
Modeling inherited metabolic disorders of the liver using human induced pluripotent stem cellsS Tamir Rashid
Laboratory for Regenerative Medicine, Cambridge Institute for Medical Research, University of Cambridge, Cambridge, UK
J Clin Invest 120:3127-36. 2010..These patient-derived hepatocytes demonstrate that it is possible to model diseases whose phenotypes are caused by pathological dysregulation of key processes within adult cells...
Disease-related amyloidogenic variants of human lysozyme trigger the unfolded protein response and disturb eye development in Drosophila melanogasterJanet R Kumita
Department of Chemistry, University of Cambridge, Cambridge, UK
FASEB J 26:192-202. 2012....
Drosophila necrotic mutations mirror disease-associated variants of human serpinsClare Green
Department of Genetics, University of Cambridge, Downing Street, Cambridge CB2 3EH, UK
Development 130:1473-8. 2003..Taken together, these data establish Drosophila as a powerful system to study serpin polymerization in vivo...
Alpha(1)-antitrypsin deficiency, liver disease and emphysemaHelen Parfrey
Department of Medicine, University of Cambridge, Cambridge Institute for Medical Research, Wellcome Trust MRC Building, Hills Road, Cambridge CB2 2XY, UK
Int J Biochem Cell Biol 35:1009-14. 2003..We show how this knowledge has led to the development of novel therapeutic approaches to treat this condition...
α(1)-antitrypsin deficiency and inflammationUgo I Ekeowa
Department of Medicine, University of Cambridge, Cambridge Institute for Medical Research, Wellcome Trust MRC Building, Cambridge, CB2 0XY, UK
Expert Rev Clin Immunol 7:243-52. 2011..Therapeutic strategies are now being developed to block the aberrant conformational transitions of mutant α(1)-antitrypsin and so treat the associated disease...
How small peptides block and reverse serpin polymerisationAiwu Zhou
Departments of Haematology and Medicine, University of Cambridge, Cambridge Institute for Medical Research, Hills Road, Cambridge CB2 2XY, UK
J Mol Biol 342:931-41. 2004..This is demonstrated here with a tetrapeptide that preferentially blocks the polymerisation of a pathologically unstable serpin commonly present in people of European descent...
Fenton chemistry and oxidative stress mediate the toxicity of the beta-amyloid peptide in a Drosophila model of Alzheimer's diseaseThomas Rival
Department of Medicine, University of Cambridge, Cambridge Institute for Medical Research, Wellcome Trust MRC Building, Cambridge, UK
Eur J Neurosci 29:1335-47. 2009....
Metabolic profiling detects biomarkers of protein degradation in COPD patientsBaljit K Ubhi
Dept of Biochemistry and Cambridge Systems Biology Centre, University of Cambridge, Cambridge, UK
Eur Respir J 40:345-55. 2012..Increased protein turnover occurred in all COPD patients with increased protein degradation in individuals with emphysema and cachexia...
Polymers of alpha(1)-antitrypsin are chemotactic for human neutrophils: a new paradigm for the pathogenesis of emphysemaJasvir S Parmar
Respiratory Medicine Division, Department of Medicine, University of Cambridge School of Clinical Medicine, Addenbrooke's and Papworth Hospitals, United Kingdom
Am J Respir Cell Mol Biol 26:723-30. 2002....
Evaluation of full-length, cleaved and nitrosylated serum surfactant protein D as biomarkers for COPDAnnelyse Duvoix
Department of Medicine, University of Cambridge, Cambridge Institute for Medical Research, Wellcome Trust MRC Building, Cambridge, UK
COPD 8:79-95. 2011..Moreover, levels of serum nitrosylated-SP-D did not correlate with serum level of SP-D or any clinical phenotype of COPD. The measurement of modified SP-D is of limited value in characterising individuals with COPD...
A Drosophila model of Alzheimer's diseaseDamian C Crowther
Department of Medicine, University of Cambridge, CIMR Wellcome Trust, Cambridge, United Kingdom CB2 2XY
Methods Enzymol 412:234-55. 2006..Phenotypes may be modulated by changes in gene expression as part of a genetic screen or by potential therapeutic compounds...
Therapeutic targets from a Drosophila model of Alzheimer's diseaseDamian C Crowther
Department of Medicine, University of Cambridge, Cambridge Institute for Medical Research, Wellcome Trust MRC Building, Hills Road, Cambridge CB2 2XY, UK
Curr Opin Pharmacol 4:513-6. 2004..Several groups are now using the power and speed of genetic screens in the fly to accelerate the discovery of novel therapeutic agents...
Airway wall thickening and emphysema show independent familial aggregation in chronic obstructive pulmonary diseaseBipen D Patel
Department of Medicine, University of Cambridge, Cambridge, UK
Am J Respir Crit Care Med 178:500-5. 2008..It is unclear whether airway wall thickening and emphysema make independent contributions to airflow limitation in chronic obstructive pulmonary disease (COPD) and whether these phenotypes cluster within families...
Systematic in vivo analysis of the intrinsic determinants of amyloid Beta pathogenicityLeila M Luheshi
Department of Chemistry, University of Cambridge, Cambridge, United Kingdom
PLoS Biol 5:e290. 2007....
The molecular aetiology of the serpinopathiesMark J Davies
Department of Medicine, University of Cambridge, Cambridge Institute for Medical Research, Wellcome Trust MRC Building, Hills Road, Cambridge CB2 0XY, United Kingdom
Int J Biochem Cell Biol 40:1273-86. 2008..We review here the structural basis of the serpinopathies and discuss how the ordered accumulation of polymers causes cell death...
Association of alpha(1)-antichymotrypsin deficiency with milder lung disease in patients with cystic fibrosisR Mahadeva
Respiratory Medicine Unit, Department of Medicine and Department of Haematology, University of Cambridge, Wellcome Trust Centre for Molecular Mechanisms in Disease, Cambridge Institute for Medical Research, Cambridge CB2 2XY, UK
Thorax 56:53-8. 2001....
Inhibitory conformation of the reactive loop of alpha 1-antitrypsinP R Elliott
Department of Haematology, University of Cambridge, UK
Nat Struct Biol 3:676-81. 1996..The beta-pleated strand conformation of the loop also accounts for the polymerization of the serpins in disease and for their association with other beta-sheet structures, most notably the beta-amyloid of Alzheimer's disease...
Polymerization of plasminogen activator inhibitor-1A Zhou
Department of Haematology, University of Cambridge, Wellcome Trust Centre for Molecular Mechanisms in Disease, Cambridge Institute for Medical Research, Wellcome Trust MRC Building, Hills Road, Cambridge, CB2 2XY, United Kingdom
J Biol Chem 276:9115-22. 2001..Glycosylated native and latent PAI-1 can also form polymers under similar conditions, which may be of in vivo importance in the low pH environment of the platelet...
Alpha-1-antitrypsin deficiency, the serpinopathies and conformational diseaseJ S Parmar
Cambridge Institute for Medical Research
J R Coll Physicians Lond 34:295-300. 2000..The interaction provides a useful paradigm for other 'conformational diseases' such as Huntington's disease, Creutzfeldt-Jakob disease and the amyloidoses...
Childhood smoking is an independent risk factor for obstructive airways disease in womenB D Patel
Department of Public Health and Primary Care, Institute of Public Health, University Forvie Site, Robinson Way, Cambridge CB2 2SR, UK
Thorax 59:682-6. 2004..To assess whether starting to smoke in childhood increases the risk of obstructive airways disease (OAD) in adult life...
Heteropolymerization of S, I, and Z alpha1-antitrypsin and liver cirrhosisR Mahadeva
Respiratory Medicine Unit, Department of Medicine and Department of Haematology, University of Cambridge, Cambridge Institute for Medical Research, Wellcome Trust MRC Building, Hills Road, Cambridge CB2 2XY, United Kingdom
J Clin Invest 103:999-1006. 1999..Taken together, these results indicate that not only may mixed variants form heteropolymers, but that this can causally lead to the development of cirrhosis...
Intraneuronal Abeta, non-amyloid aggregates and neurodegeneration in a Drosophila model of Alzheimer's diseaseD C Crowther
Department of Medicine, University of Cambridge, Cambridge Institute for Medical Research, Wellcome Trust MRC Building, Hills Road, Cambridge CB2 2XY, UK
Neuroscience 132:123-35. 2005..Moreover it provides a platform to dissect the pathways of neurodegeneration in Alzheimer's disease and to develop novel therapeutic interventions...
A kinetic mechanism for the polymerization of alpha1-antitrypsinT R Dafforn
Department of Haematology, Department of Medicine, University of Cambridge, Cambridge Institute for Medical Research, Wellcome Trust Medical Research Council Building, Hills Road, Cambridge CB2 2XY, United Kingdom
J Biol Chem 274:9548-55. 1999..Taken together, these data allow us to propose a kinetic mechanism for alpha1-antitrypsin polymer formation that involves the generation of an unstable intermediate, which can form polymers or generate latent protein...
Neuroserpin: a serpin to think aboutE Miranda
Department of Medicine, University of Cambridge, Cambridge Institute for Medical Research, Wellcome Trust Medical Research Council Building, Hills Road, Cambridge CB2 2XY, United Kingdom
Cell Mol Life Sci 63:709-22. 2006..We review here the role of neuroserpin and other proteinase inhibitors in brain development, function and disease...
Wild-type alpha 1-antitrypsin is in the canonical inhibitory conformationP R Elliott
Department of Medicine, University of Cambridge, UK
J Mol Biol 275:419-25. 1998..This pocket may provide a target for rational drug design to prevent the formation of polymers and the associated plasma deficiency, liver cirrhosis and emphysema...
What can naturally occurring mutations tell us about the pathogenesis of COPD?S J Marciniak
Department of Medicine, University of Cambridge, Cambridge Institute for Medical Research, Wellcome Trust MRC Building, Hills Road, Cambridge CB2 0XY, UK
Thorax 64:359-64. 2009..Only then will we have new insights into the pathways that underlie this common condition...
Topography of a 2.0 A structure of alpha1-antitrypsin reveals targets for rational drug design to prevent conformational diseaseP R Elliott
Department of Medicine, University of Cambridge, The Wellcome Trust Centre for Molecular Mechanisms in Disease, Cambridge Institute for Medical Research, United Kingdom
Protein Sci 9:1274-81. 2000..This structure allows us to define five cavities that are potential targets for rational drug design to develop agents that will prevent conformational transitions and ameliorate the associated disease...
Inactive conformation of the serpin alpha(1)-antichymotrypsin indicates two-stage insertion of the reactive loop: implications for inhibitory function and conformational diseaseB Gooptu
Respiratory Medicine Unit, Department of Medicine University of Cambridge, United Kingdom
Proc Natl Acad Sci U S A 97:67-72. 2000....
Siblings of patients with severe chronic obstructive pulmonary disease have a significant risk of airflow obstructionS C McCloskey
Respiratory Medicine Unit, Department of Medicine, University of Cambridge, The Wellcome Trust Centre for Molecular Mechanisms in Disease, Cambridge Institute for Medical Research, Cambridge, United Kingdom
Am J Respir Crit Care Med 164:1419-24. 2001..39 (95% confidence interval, 2.49 to 11.67) when compared with matched control subjects. Taken together these results demonstrate a significant familial risk of airflow obstruction in smoking siblings of patients with severe COPD...
Implications for function and therapy of a 2.9 A structure of binary-complexed antithrombinR Skinner
Department of Haematology, University of Cambridge, Hills Road, Cambridge, CB2 2QH, UK
J Mol Biol 283:9-14. 1998....
Smoking related COPD and facial wrinkling: is there a common susceptibility?B D Patel
Department of Public Health and Primary Care, University of Cambridge, UK
Thorax 61:568-571. 2006..We have tested the hypothesis that there is a common susceptibility for the development of COPD and facial wrinkling in cigarette smokers...
The effect of sequence variations within the coding region of the C1 inhibitor gene on disease expression and protein function in families with hereditary angio-oedemaS-A Cumming
Respiratory Medicine Unit, Department of Medicine, University of Cambridge, Cambridge Institute for Medical Research, Wellcome Trust/MRC Building, Hills Road, Cambridge CB2 2XY, UK
J Med Genet 40:e114. 2003
Dietary antioxidants and asthma in adultsB D Patel
Department of Public Health and Primary Care, Institute of Public Health, Cambridge, UK
Thorax 61:388-93. 2006..These findings suggest that diet may be a potentially modifiable risk factor for the development of asthma...
Polymers and inflammation: disease mechanisms of the serpinopathiesBibek Gooptu
School of Crystallography, Birkbeck College, University of London, London WC1E 7HX, England, UK
J Exp Med 205:1529-34. 2008..Here, we describe how these three mechanisms can be integrated into a new model of the pathogenesis of emphysema caused by mutations in the serpin alpha1-antitrypsin...
Differential detection of PAS-positive inclusions formed by the Z, Siiyama, and Mmalton variants of alpha1-antitrypsinSabina Janciauskiene
Department of Medicine, University Hospital Malmo, Malmo, Sweden
Hepatology 40:1203-10. 2004..In conclusion, the ATZ11 monoclonal antibody detects Z alpha(1)-antitrypsin in hepatic inclusions by detecting a neoepitope that is specific to the polymeric conformer and that is localized close to residue 342...
Promiscuous beta-strand interactions and the conformational diseasesMichelle K M Chow
Department of Biochemistry and Molecular Biology, Monash University, VIC 3800, Australia
Curr Med Chem 11:491-9. 2004..We will discuss the successes and pitfalls of such approaches to demonstrate how similar approaches may be applied to any misfolding protein...
Expression of the serine protease inhibitor neuroserpin in cells of the human myeloid lineageSarah A Kennedy
School of Biological Sciences, Level 4, Thomas Building, University of Auckland, Private Bag 92019, Auckland 1010, New Zealand
Thromb Haemost 97:394-9. 2007..This analysis revealed differential expression of PAI-1 and neuroserpin suggesting they may have different functions in human immune cells...
Association between conformational mutations in neuroserpin and onset and severity of dementiaRichard L Davis
Department of Pathology, Upstate Medical University, Syracuse, NY, USA
Lancet 359:2242-7. 2002..To clarify this issue, we investigated five families with typical neuroserpin inclusion bodies but with various neurological manifestations...
Serpin identification, production, and characterizationGary A Silverman
Methods 32:71-2. 2004
The SERPINE2 gene is associated with chronic obstructive pulmonary disease in two large populationsGuohua Zhu
GlaxoSmithKline R and D, 5 Moore Drive, Research Triangle Park, NC 27709, USA
Am J Respir Crit Care Med 176:167-73. 2007....
