Jerry R Mendell

Summary

Affiliation: The Ohio State University
Country: USA

Publications

  1. ncbi request reprint Novel compound heterozygous laminina2-chain gene (LAMA2) mutations in congenital muscular dystrophy. Mutations in brief no. 159. Online
    J T Mendell
    Department of Biochemistry, Ohio State University, 1654 Upham Drive, Columbus 43210, Ohio, USA
    Hum Mutat 12:135. 1998
  2. ncbi request reprint Clinical practice. Painful sensory neuropathy
    Jerry R Mendell
    Department of Neurology, Ohio State University, Columbus 43210, USA
    N Engl J Med 348:1243-55. 2003
  3. ncbi request reprint Gene transfer for neurologic disease: agencies, policies, and process
    Jerry R Mendell
    Center for Neuromuscular Disorders, Children s Research Institute, Department of Neurology, The Ohio State University, WA 3024, 700 Children s Drive, Columbus, OH 43205, USA
    Neurology 63:2225-32. 2004
  4. pmc Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D
    Jerry R Mendell
    Department of Pediatrics, Ohio State University, Columbus, OH, USA
    Ann Neurol 68:629-38. 2010
  5. pmc Gene therapy for muscular dystrophy: lessons learned and path forward
    Jerry R Mendell
    Center for Gene Therapy, Research Institute at Nationwide Children s Hospital, Department of Pediatrics, The Ohio State University, Columbus, OH 43205, USA
    Neurosci Lett 527:90-9. 2012
  6. pmc Homologous recombination mediates functional recovery of dysferlin deficiency following AAV5 gene transfer
    William E Grose
    Department of Pediatrics, The Ohio State University, Columbus, Ohio, United States of America
    PLoS ONE 7:e39233. 2012
  7. pmc Novel diagnostic features of dysferlinopathies
    Xiomara Q Rosales
    Department of Pediatrics, Neuromuscular Division, Nationwide Children s Hospital, Columbus, Ohio, USA
    Muscle Nerve 42:14-21. 2010
  8. doi request reprint Gentamicin-induced readthrough of stop codons in Duchenne muscular dystrophy
    Vinod Malik
    Center for Gene Therapy, Research Institute at Nationwide Children s Hospital, Ohio State University, Columbus, OH 43205, USA
    Ann Neurol 67:771-80. 2010
  9. doi request reprint AAV-mediated gene therapy to the isolated limb in rhesus macaques
    Louise R Rodino-Klapac
    Center for Gene Therapy, The Research Institute at Nationwide Children s Hospital and Department of Pediatrics, The Ohio State University, Columbus, OH, USA
    Methods Mol Biol 709:287-98. 2011
  10. pmc Follistatin gene delivery enhances muscle growth and strength in nonhuman primates
    Janaiah Kota
    Center for Gene Therapy, Research Institute at Nationwide Children s Hospital, Columbus, OH 43205, USA
    Sci Transl Med 1:6ra15. 2009

Detail Information

Publications51

  1. ncbi request reprint Novel compound heterozygous laminina2-chain gene (LAMA2) mutations in congenital muscular dystrophy. Mutations in brief no. 159. Online
    J T Mendell
    Department of Biochemistry, Ohio State University, 1654 Upham Drive, Columbus 43210, Ohio, USA
    Hum Mutat 12:135. 1998
    ..By either mechanism the phenotype of congenital muscular dystrophy is believed to be the result of disruption of linkage between the extracellular matrix and the dystrophin glycoprotein complex...
  2. ncbi request reprint Clinical practice. Painful sensory neuropathy
    Jerry R Mendell
    Department of Neurology, Ohio State University, Columbus 43210, USA
    N Engl J Med 348:1243-55. 2003
  3. ncbi request reprint Gene transfer for neurologic disease: agencies, policies, and process
    Jerry R Mendell
    Center for Neuromuscular Disorders, Children s Research Institute, Department of Neurology, The Ohio State University, WA 3024, 700 Children s Drive, Columbus, OH 43205, USA
    Neurology 63:2225-32. 2004
    ..The links provided to all appropriate Web sites will facilitate the process for the clinician investigator...
  4. pmc Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D
    Jerry R Mendell
    Department of Pediatrics, Ohio State University, Columbus, OH, USA
    Ann Neurol 68:629-38. 2010
    ....
  5. pmc Gene therapy for muscular dystrophy: lessons learned and path forward
    Jerry R Mendell
    Center for Gene Therapy, Research Institute at Nationwide Children s Hospital, Department of Pediatrics, The Ohio State University, Columbus, OH 43205, USA
    Neurosci Lett 527:90-9. 2012
    ..Increasing the size and strength of the muscle is the goal of this study. Most importantly, no adverse events have been encountered in any of these clinical trials...
  6. pmc Homologous recombination mediates functional recovery of dysferlin deficiency following AAV5 gene transfer
    William E Grose
    Department of Pediatrics, The Ohio State University, Columbus, Ohio, United States of America
    PLoS ONE 7:e39233. 2012
    ..We provide proof of principle that AAV5 mediated delivery of dysferlin is a highly promising strategy for treatment of dysferlinopathies and has far-reaching implications for the therapeutic delivery of other large genes...
  7. pmc Novel diagnostic features of dysferlinopathies
    Xiomara Q Rosales
    Department of Pediatrics, Neuromuscular Division, Nationwide Children s Hospital, Columbus, Ohio, USA
    Muscle Nerve 42:14-21. 2010
    ..Correlative studies showed colocalization of amyloid with deposition of dysferlin. The present data further serve to guide clinicians facing the expensive task of molecular characterization of patients with an LGMD phenotype...
  8. doi request reprint Gentamicin-induced readthrough of stop codons in Duchenne muscular dystrophy
    Vinod Malik
    Center for Gene Therapy, Research Institute at Nationwide Children s Hospital, Ohio State University, Columbus, OH 43205, USA
    Ann Neurol 67:771-80. 2010
    ..Mutation suppression of stop codons, successfully achieved in the mdx mouse using gentamicin, represents an important evolving treatment strategy in Duchenne muscular dystrophy (DMD)...
  9. doi request reprint AAV-mediated gene therapy to the isolated limb in rhesus macaques
    Louise R Rodino-Klapac
    Center for Gene Therapy, The Research Institute at Nationwide Children s Hospital and Department of Pediatrics, The Ohio State University, Columbus, OH, USA
    Methods Mol Biol 709:287-98. 2011
    ..We also provide methods for assessing transduction efficiency of microdystrophin.FLAG following the IFLP vascular delivery protocol...
  10. pmc Follistatin gene delivery enhances muscle growth and strength in nonhuman primates
    Janaiah Kota
    Center for Gene Therapy, Research Institute at Nationwide Children s Hospital, Columbus, OH 43205, USA
    Sci Transl Med 1:6ra15. 2009
    ..Our results, together with the findings in mice, suggest that therapy with AAV1-FS344 may improve muscle mass and function in patients with certain degenerative muscle disorders...
  11. pmc Persistent expression of FLAG-tagged micro dystrophin in nonhuman primates following intramuscular and vascular delivery
    Louise R Rodino-Klapac
    Department of Pediatrics, The Ohio State University Nationwide Children s Hospital, Columbus, Ohio 43205, USA
    Mol Ther 18:109-17. 2010
    ..In summary, an epitope-tagged micro-dystrophin cassette enhances the ability to evaluate site-specific localization and distribution of gene expression in the NHP in preparation for vascular delivery clinical trials...
  12. pmc Overexpression of Galgt2 in skeletal muscle prevents injury resulting from eccentric contractions in both mdx and wild-type mice
    Paul T Martin
    Center for Gene Therapy, The Research Institute at Nationwide Children s Hospital, Department of Pediatrics, The Ohio State Univ College of Medicine, 304 Hamilton Hall, 1645 Neil Ave, Columbus, OH 43210 1218, USA
    Am J Physiol Cell Physiol 296:C476-88. 2009
    ..That overexpression also prevents loss of force in nondystrophic muscles suggests that Galgt2 is a therapeutic target with broad potential applications...
  13. pmc Dystrophin immunity in Duchenne's muscular dystrophy
    Jerry R Mendell
    Center for Gene Therapy, Research Institute at Nationwide Children s Hospital, Columbus, OH 43205, USA
    N Engl J Med 363:1429-37. 2010
    ..Funded by the Muscular Dystrophy Association and others; ClinicalTrials.gov number, NCT00428935.)...
  14. pmc Impaired regeneration in LGMD2A supported by increased PAX7-positive satellite cell content and muscle-specific microrna dysregulation
    Xiomara Q Rosales
    Neuromuscular Center at The Research Institute at Nationwide Children s Hospital, Columbus, Ohio Department of Pediatrics and Center for Gene Therapy, The Research Institute at Nationwide Children s Hospital, 700 Children s Drive, Columbus, Ohio, 43205 The Ohio State University, Columbus, Ohio
    Muscle Nerve 47:731-9. 2013
    ..This results in impaired regeneration and fibrosis. Muscle Nerve 47: 731-739, 2013...
  15. pmc Inhibition of myostatin with emphasis on follistatin as a therapy for muscle disease
    Louise R Rodino-Klapac
    Center for Gene Therapy, Research Institute at Nationwide Children s Hospital, 700 Children s Drive, Columbus, Ohio 43205 USA
    Muscle Nerve 39:283-96. 2009
    ..These findings provide the impetus to move toward gene therapy clinical trials with delivery of AAV-FS344 to increase size and function of muscle in patients with neuromuscular disease...
  16. doi request reprint Limb-girdle muscular dystrophy type 2D gene therapy restores alpha-sarcoglycan and associated proteins
    Jerry R Mendell
    Department of Pediatrics, Ohio State University, Columbus, OH, USA
    Ann Neurol 66:290-7. 2009
    ..Gene replacement represents a strategy for correcting the underlying defect. Questions related to this approach were addressed in this clinical trial, particularly the need for immunotherapy and persistence of gene expression...
  17. doi request reprint Knee extensor strength exhibits potential to predict function in sporadic inclusion-body myositis
    Linda Pax Lowes
    Center for Gene Therapy, Nationwide Children s Hospital, 700 Children s Drive, Columbus, Ohio 43205, USA
    Muscle Nerve 45:163-8. 2012
    ..This has immediate relevance to translational studies that attempt to improve quadriceps strength in sporadic inclusion-body myositis (sIBM)...
  18. doi request reprint Effects of angiotensin-converting enzyme inhibitors and/or beta blockers on the cardiomyopathy in Duchenne muscular dystrophy
    Laurence Viollet
    Nationwide Children s Hospital, Columbus, OH, USA
    Am J Cardiol 110:98-102. 2012
    ..No significant difference occurred in EF improvement between treatment groups. In conclusion, treatment with ACE inhibitor or ACE inhibitor plus BB can delay progression of cardiomyopathy...
  19. pmc A translational approach for limb vascular delivery of the micro-dystrophin gene without high volume or high pressure for treatment of Duchenne muscular dystrophy
    Louise R Rodino-Klapac
    Center for Gene Therapy, Columbus Children s Research Institute, Columbus Children s Hospital, 700 Children s Dr, Columbus, Ohio 43205, USA
    J Transl Med 5:45. 2007
    ....
  20. pmc Analysis of dystrophin deletion mutations predicts age of cardiomyopathy onset in becker muscular dystrophy
    Rita Wen Kaspar
    Center for Gene Therapy, The Research Institute at Nationwide Children s Hospital College of Nursing, The Ohio State University, Columbus, Ohio, USA
    Circ Cardiovasc Genet 2:544-51. 2009
    ..This approach was chosen to connect dystrophin structure with function in the heart...
  21. pmc Essential metabolic, anti-inflammatory, and anti-tumorigenic functions of miR-122 in liver
    Shu Hao Hsu
    Molecular, Cellular, and Developmental Biology Program, The Ohio State University, Columbus, OH, USA
    J Clin Invest 122:2871-83. 2012
    ....
  22. pmc Systemic gene delivery in large species for targeting spinal cord, brain, and peripheral tissues for pediatric disorders
    Adam K Bevan
    Center for Gene Therapy, The Research Institute at Nationwide Children s Hospital, Columbus, Ohio 43205, USA
    Mol Ther 19:1971-80. 2011
    ..Our findings support the use of AAV9 for gene transfer to the CNS for disorders in pediatric populations...
  23. pmc RNA interference inhibits DUX4-induced muscle toxicity in vivo: implications for a targeted FSHD therapy
    Lindsay M Wallace
    Molecular, Cellular, and Developmental Biology Graduate Program, The Ohio State University, Columbus, Ohio, USA
    Mol Ther 20:1417-23. 2012
    ..We found that adeno-associated viral (AAV) vector-delivered therapeutic microRNAs corrected DUX4-associated myopathy in mouse muscle. These results provide proof-of-principle for RNAi therapy of FSHD through DUX4 inhibition...
  24. pmc Cardiovascular magnetic resonance of cardiomyopathy in limb girdle muscular dystrophy 2B and 2I
    Xiomara Q Rosales
    Center for Gene Therapy, The Research Institute at Nationwide Children s Hospital, Columbus, Ohio 43205, USA
    J Cardiovasc Magn Reson 13:39. 2011
    ....
  25. pmc Long-term enhancement of skeletal muscle mass and strength by single gene administration of myostatin inhibitors
    Amanda M Haidet
    The Research Institute, Nationwide Children s Hospital, Columbus, OH 43205, USA
    Proc Natl Acad Sci U S A 105:4318-22. 2008
    ..These results demonstrate a promising therapeutic strategy that warrants consideration for clinical trials in human muscle diseases...
  26. doi request reprint Cardiac management in neuromuscular diseases
    Hugh D Allen
    The Ohio State University College of Medicine, Columbus, OH, USA
    Phys Med Rehabil Clin N Am 23:855-68. 2012
    ..Some dystrophies can have significant conduction abnormalities requiring pacemaker treatment. Others with ventricular tachydysrhythmias may necessitate internal cardiac defibrillator placement...
  27. pmc Nonsense mutation-associated Becker muscular dystrophy: interplay between exon definition and splicing regulatory elements within the DMD gene
    Kevin M Flanigan
    Center for Gene Therapy, Nationwide Children s Hospital, Columbus, Ohio, USA
    Hum Mutat 32:299-308. 2011
    ..We present a new model based on the combination of exon definition and intronic splicing regulatory elements for the selective association of BMD nonsense mutations with a subset of DMD exons prone to mutation-induced exon skipping...
  28. doi request reprint Evidence-based path to newborn screening for Duchenne muscular dystrophy
    Jerry R Mendell
    Department of Pediatrics, Ohio State University and Nationwide Children s Hospital, Columbus, OH 43205, USA
    Ann Neurol 71:304-13. 2012
    ..As a marker for newborn screening, CK in Duchenne muscular dystrophy (DMD) results in false-positive testing. In this report, we introduce a 2-tier system using the dried blood spot to first assess CK with follow-up DMD gene testing...
  29. pmc Therapeutic microRNA delivery suppresses tumorigenesis in a murine liver cancer model
    Janaiah Kota
    Center for Gene Therapy, The Research Institute at Nationwide Children s Hospital, Columbus, OH 43205, USA
    Cell 137:1005-17. 2009
    ..These findings suggest that delivery of miRNAs that are highly expressed and therefore tolerated in normal tissues but lost in disease cells may provide a general strategy for miRNA replacement therapies...
  30. doi request reprint Fidelity of gamma-glutamyl transferase (GGT) in differentiating skeletal muscle from liver damage
    Xiomara Q Rosales
    Department of Pediatrics, The Ohio State University, Columbus, Ohio, USA
    J Child Neurol 23:748-51. 2008
    ..Validation of this finding is essential for management of patients with muscle disorders exposed to potentially hepatotoxic drugs for clinical management or monitoring subjects participating in clinical trials...
  31. ncbi request reprint Lambert-Eaton myasthenic syndrome in children
    Chang Yong Tsao
    Department of Pediatrics and Neurology, College of Medicine and Public Health, The Ohio State University, USA
    J Child Neurol 17:74-6. 2002
    ..High-frequency repetitive nerve stimulation and P/Q-type calcium-channel antibodies may confirm the diagnosis...
  32. ncbi request reprint Dermatomyositis in two siblings and a brief review of familial dermatomyositis
    Chang Yong Tsao
    Department of Pediatrics, The Ohio State University, Columbus, USA
    J Child Neurol 17:540-2. 2002
    ..Familial dermatomyositis can occur in different family members, and even dermatomyositis and polymyositis can coexist in the same family...
  33. ncbi request reprint Combined partial deficiencies of carnitine palmitoyltransferase II and mitochondrial complex I presenting as increased serum creatine kinase level
    Chang Yong Tsao
    Department of Pediatrics and Neurology, College of Medicine and Public Health, The Ohio State University, Columbus, USA
    J Child Neurol 17:304-6. 2002
    ..Metabolic myopathy may present with chronic fatigue and a persistently high serum creatine kinase level but without muscle weakness and may be attributable to combined enzyme defects...
  34. doi request reprint Update on the treatment of Duchenne muscular dystrophy
    Louise R Rodino-Klapac
    Department of Pediatrics, The Ohio State University, and Nationwide Children s Hospital, Columbus, OH 43210, USA
    Curr Neurol Neurosci Rep 13:332. 2013
    ..The advantages of each approach and challenges in translation are outlined in detail. Individually or in combination, all of these therapeutic strategies hold great promise for treatment of this devastating childhood disease...
  35. pmc AAV-mediated Overexpression of Human α7 Integrin Leads to Histological and Functional Improvement in Dystrophic Mice
    Kristin N Heller
    1 Molecular, Cellular, and Developmental Biology Graduate Program, The Ohio State University, Columbus, Ohio, USA 2 Center for Gene Therapy, The Research Institute at Nationwide Children s Hospital, Columbus, Ohio, USA
    Mol Ther 21:520-5. 2013
    ..This therapeutic approach demonstrates promise as a viable treatment for DMD with further implications for other forms of muscular dystrophy...
  36. ncbi request reprint Gene therapy for duchenne muscular dystrophy: expectations and challenges
    Louise R Rodino-Klapac
    Center for Gene Therapy, Columbus Children s Research Institute, Columbus, OH, USA
    Arch Neurol 64:1236-41. 2007
    ..This article highlights the challenges and potential pitfalls as the field advances this treatment modality to clinical reality...
  37. pmc Molecular therapeutic strategies targeting Duchenne muscular dystrophy
    Jerry R Mendell
    Center for Gene Therapy, Nationwide Children s Hospital, Department of Pediatrics, The Ohio State University, Columbus, OH 43205, USA
    J Child Neurol 25:1145-8. 2010
    ..The results are modest and encumbered by side effects. The authors review 3 molecular therapeutic approaches that have been introduced into the clinic: (1) gene replacement therapy, (2) mutation suppression, and (3) exon skipping...
  38. doi request reprint The muscular dystrophies: distinct pathogenic mechanisms invite novel therapeutic approaches
    Zarife Sahenk
    The Research Institute at Nationwide Children s Hospital, Departments of Pediatrics and Neurology, Ohio State University, 700 Children s Drive, Room WA3024, Columbus, OH 43205, USA
    Curr Rheumatol Rep 13:199-207. 2011
    ..In many ways, the molecular gene defects are the most traditional. Gene repair strategies have advanced to the level of clinical testing, and we hope they will provide relief for this most devastating form of muscular dystrophy...
  39. pmc Aminoglycoside-induced mutation suppression (stop codon readthrough) as a therapeutic strategy for Duchenne muscular dystrophy
    Vinod Malik
    The Research Institute at Nationwide Children s Hospital and Department of Pediatrics at The Ohio State University College of Medicine, Columbus, OH, USA
    Ther Adv Neurol Disord 3:379-89. 2010
    ..Here we review nonsense mutation suppression by aminoglycosides as a therapeutic strategy to treat DMD with special emphasis on gentamicin-induced readthrough of disease-causing premature termination codons...
  40. pmc Utility of cystatin C to monitor renal function in Duchenne muscular dystrophy
    Laurence Viollet
    Research Institute at Nationwide Children s Hospital and Department of Pediatrics at Ohio State University, 700 Children s Drive, Room 3011, Columbus, Ohio 43205, USA
    Muscle Nerve 40:438-42. 2009
    ..01). In one DMD subject in renal failure, cystatin C was elevated. This study demonstrates the potential value of cystatin C as a biomarker for monitoring renal function in DMD. Its applicability extends to other neuromuscular diseases...
  41. doi request reprint Correlation of knee strength to functional outcomes in Becker muscular dystrophy
    Lindsay N Alfano
    Center for Gene Therapy and the Paul D Wellstone Cooperative Research Center, The Research Institute at Nationwide Children s Hospital, 700 Children s Drive, Columbus, Ohio 43205, USA
    Muscle Nerve 47:550-4. 2013
    ....
  42. pmc Emerging drugs for Duchenne muscular dystrophy
    Vinod Malik
    The Ohio State University, Research Institute, Nationwide Children s Hospital and, Department of Pediatrics, Columbus, OH 43205, USA
    Expert Opin Emerg Drugs 17:261-77. 2012
    ..The review emphasizes that the goal of treatment should be to find a product at least as good as glucocorticoids with a lower side effect profile or with a significant glucocorticoid sparing effect...
  43. doi request reprint A phase I/IItrial of MYO-029 in adult subjects with muscular dystrophy
    Kathryn R Wagner
    Department of Neurology, The Johns Hopkins University School of Medicine, Baltimore, MD 21287 7519, USA
    Ann Neurol 63:561-71. 2008
    ..We conducted a safety trial of a neutralizing antibody to myostatin, MYO-029, in adult muscular dystrophies (Becker muscular dystrophy, facioscapulohumeral dystrophy, and limb-girdle muscular dystrophy)...
  44. ncbi request reprint Challenges in drug development for muscle disease: a stakeholders' meeting
    Jerry R Mendell
    Columbus Children s Research Institute, and Ohio State University, 700 Children s Drive, Columbus, OH 43235, USA
    Muscle Nerve 35:8-16. 2007
    ..The meeting provided a format for communication for diverse disciplines that usually have no common meeting ground, helping to lay the foundation for bringing products to market in a timely fashion...
  45. doi request reprint Re-examination of the electrocardiogram in boys with Duchenne muscular dystrophy and correlation with its dilated cardiomyopathy
    Philip T Thrush
    Department of Pediatrics, The Ohio State University, and the Heart Center, Nationwide Children s Hospital, Columbus, Ohio, USA
    Am J Cardiol 103:262-5. 2009
    ..Previously reported characteristic ECG changes are seen in a minority of DMD cases. The most common findings are short PR interval and RVH. Prominent Q waves in leads II, III, aVF, V5, and V6 are more likely...
  46. doi request reprint Report of MDA muscle disease symposium on newborn screening for Duchenne muscular dystrophy
    Jerry R Mendell
    Nationwide Children s Hospital, Paul D Wellstone Muscular Dystrophy Cooperative Research Center and Department of Pediatrics, Ohio State University, 700 Childrens Drive, Columbus, Ohio, 43205, USA
    Muscle Nerve 48:21-6. 2013
    ..Conclusions from this symposium with supportive data could have a significant impact on propelling efforts for approval of newborn screening for DMD...
  47. ncbi request reprint Challenges for gene therapy for muscular dystrophy
    Jerry R Mendell
    Center for Gene Therapy, Columbus Children s Research Institute, Columbus, OH 43205, USA
    Curr Neurol Neurosci Rep 6:47-56. 2006
    ..This review examines recent progress and the hurdles remaining to achieve gene-based treatment therapies for muscular dystrophy...
  48. doi request reprint An analysis of disease severity based on SMN2 copy number in adults with spinal muscular atrophy
    Bakri Elsheikh
    Department of Neurology, Ohio State University, 421 Means Hall, 1654 Upham Drive, Columbus, Ohio 43210, USA
    Muscle Nerve 40:652-6. 2009
    ..There was, however, no difference between the groups in quantitative muscle strength or pulmonary function testing. Functional scale may be a more discriminating outcome measure for SMA clinical trials...
  49. pmc The congenital muscular dystrophies: recent advances and molecular insights
    Jerry R Mendell
    Department of Pediatrics, Columbus Children s Hospital and Research Institute and The Ohio State University, 700 Children s Drive, Columbus, OH 43205, USA
    Pediatr Dev Pathol 9:427-43. 2006
    ..g., family history, central nervous system features) can help guide the battery of immunostains necessary to target an unequivocal diagnosis...
  50. ncbi request reprint Risks, benefits, and consent in the age of gene therapy
    Jerry R Mendell
    Neurology 66:964-5. 2006
  51. pmc Gene transfer demonstrates that muscle is not a primary target for non-cell-autonomous toxicity in familial amyotrophic lateral sclerosis
    Timothy M Miller
    Ludwig Institute for Cancer Research, La Jolla, CA 92093, USA
    Proc Natl Acad Sci U S A 103:19546-51. 2006
    ..Thus, SOD1-mutant-mediated damage within muscles is not a significant contributor to non-cell-autonomous pathogenesis in ALS, and enhancing muscle mass and strength provides no benefit in slowing disease onset or progression...