genetic therapy


Summary: Techniques and strategies which include the use of coding sequences and other conventional or radical means to transform or modify cells for the purpose of treating or reversing disease conditions.

Top Publications

  1. Sheridan C. Gene therapy finds its niche. Nat Biotechnol. 2011;29:121-8 pubmed publisher
    ..Gene therapy is finally poised to make a contribution to the treatment of debilitating, highly penetrant genetic diseases that have proved intractable to other regimens. ..
  2. Naldini L. Medicine. A comeback for gene therapy. Science. 2009;326:805-6 pubmed publisher
  3. Feng Y, Leavitt M, Tritz R, Duarte E, Kang D, Mamounas M, et al. Inhibition of CCR5-dependent HIV-1 infection by hairpin ribozyme gene therapy against CC-chemokine receptor 5. Virology. 2000;276:271-8 pubmed
    ..These results suggest a novel gene therapy approach to preventing or slowing the disease progression of HIV-1 infection. ..
  4. Adair J, Kubek S, Kiem H. Hematopoietic Stem Cell Approaches to Cancer. Hematol Oncol Clin North Am. 2017;31:897-912 pubmed publisher
    ..We also discuss how HSCs can be harnessed to produce powerful tumor killing T cells, potentially benefitting and complementing T-cell-based immunotherapies. ..
  5. Kalimuthu S, Oh J, Gangadaran P, Zhu L, Lee H, Jeon Y, et al. Genetically engineered suicide gene in mesenchymal stem cells using a Tet-On system for anaplastic thyroid cancer. PLoS ONE. 2017;12:e0181318 pubmed publisher
    ..In addition, our findings provide an innovative therapeutic approach for using the Tet-On system to eradicate tumors by simple, repeated administration of MSC-Tet-TK/Fluc cells with DOX and GCV. ..
  6. Maljevic S, Reid C, Petrou S. Models for discovery of targeted therapy in genetic epileptic encephalopathies. J Neurochem. 2017;143:30-48 pubmed publisher
  7. Bahner I, Kearns K, Hao Q, Smogorzewska E, Kohn D. Transduction of human CD34+ hematopoietic progenitor cells by a retroviral vector expressing an RRE decoy inhibits human immunodeficiency virus type 1 replication in myelomonocytic cells produced in long-term culture. J Virol. 1996;70:4352-60 pubmed
    ..These findings demonstrate the ability of an RRE decoy strategy to inhibit HIV-1 replication in primary human myelomonocytic cells after transduction of CD34+ progenitor cells, without adverse effects on hematopoietic cell function. ..
  8. Byrne P, Collins S, Mah C, Smith B, Conlon T, Martin S, et al. Phase I/II trial of diaphragm delivery of recombinant adeno-associated virus acid alpha-glucosidase (rAAaV1-CMV-GAA) gene vector in patients with Pompe disease. Hum Gene Ther Clin Dev. 2014;25:134-63 pubmed publisher
  9. Munson R, Davis L. Germ-line gene therapy and the medical imperative. Kennedy Inst Ethics J. 1992;2:137-58 pubmed
    ..There is no moral reason, then, not to develop and employ germ-line gene therapy. Taking the offensive, we argue next that medicine has a prima facie moral obligation to do so. ..

More Information


  1. Dokal I. Dyskeratosis congenita in all its forms. Br J Haematol. 2000;110:768-79 pubmed
  2. Olivieri N. The beta-thalassemias. N Engl J Med. 1999;341:99-109 pubmed
  3. Barinaga M. From bench top to bedside. Science. 1997;278:1036-9 pubmed
  4. Stroes E, Nierman M, Meulenberg J, Franssen R, Twisk J, Henny C, et al. Intramuscular administration of AAV1-lipoprotein lipase S447X lowers triglycerides in lipoprotein lipase-deficient patients. Arterioscler Thromb Vasc Biol. 2008;28:2303-4 pubmed publisher
  5. Kirik D, Bjorklund A. Parkinson's disease: viral vector delivery of parkin generates model results in rats. Gene Ther. 2005;12:727-9 pubmed
  6. Biffi A. Hematopoietic Gene Therapies for Metabolic and Neurologic Diseases. Hematol Oncol Clin North Am. 2017;31:869-881 pubmed publisher
    ..Genetic engineering of the cells to be transplanted (hematopoietic stem cell) may endow the brain myeloid progeny of these cells with enhanced or novel functions, contributing to therapeutic effects. ..
  7. Liew A, O BRIEN T. Therapeutic potential for mesenchymal stem cell transplantation in critical limb ischemia. Stem Cell Res Ther. 2012;3:28 pubmed publisher
    ..Preclinical efficacy data in animal models of hindlimb ischemia, current early-phase human trial data, and considerations for future MSC-based therapy in CLI will also be discussed. ..
  8. Stuckey D. Changing the Face of Modern Medicine: Stem Cells & Gene Therapy, October 18-21, 2016, Florence, Italy. EBioMedicine. 2016;14:3-4 pubmed publisher
  9. Walther W, Schlag P. Current status of gene therapy for cancer. Curr Opin Oncol. 2013;25:659-64 pubmed
    ..The field is moving toward a therapeutic option, which will also be applicable for the treatment of disseminated metastases. Furthermore, numerous new approaches are about to be translated in clinical trials. ..
  10. Stoessl A. Gene therapy for Parkinson's disease: a step closer?. Lancet. 2014;383:1107-9 pubmed publisher
  11. Athanasopoulos T, Munye M, Yáñez Muñoz R. Nonintegrating Gene Therapy Vectors. Hematol Oncol Clin North Am. 2017;31:753-770 pubmed publisher
    ..Particular emphasis is placed on their use in hematologic disease. ..
  12. Zeng M, Szymczak M, Ahuja M, Zheng C, Yin H, Swaim W, et al. Restoration of CFTR Activity in Ducts Rescues Acinar Cell Function and Reduces Inflammation in Pancreatic and Salivary Glands of Mice. Gastroenterology. 2017;153:1148-1159 pubmed publisher
    ..Our findings reveal that correcting ductal function is sufficient to rescue acinar cell function and suggests that CFTR correctors are strong candidates for the treatment of Sjögren's syndrome and pancreatitis. ..
  13. Goyvaerts C, De Vlaeminck Y, Escors D, Lienenklaus S, Keyaerts M, Raes G, et al. Antigen-presenting cell-targeted lentiviral vectors do not support the development of productive T-cell effector responses: implications for in vivo targeted vaccine delivery. Gene Ther. 2017;24:370-375 pubmed publisher
    ..1-LVs trigger a type I interferon response with anti-viral capacity. These findings question the rationale of targeting LVs to APCs and argue for the development of VSV.G-LVs with an improved safety profile. ..
  14. Schimmer J, Breazzano S. Investor Outlook: The Unanswered Questions. Hum Gene Ther Clin Dev. 2017;28:57-61 pubmed publisher
    ..In this report, we detail the success and setbacks of 2016 and highlight the unanswered questions-and how the answers may shape the field in the years ahead. ..
  15. Chandler R, LaFave M, Varshney G, Trivedi N, Carrillo Carrasco N, Senac J, et al. Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy. J Clin Invest. 2015;125:870-80 pubmed publisher
    ..Together, our results define aspects of AAV-mediated gene therapy that influence genotoxicity and suggest that these features should be considered for design of both safer AAV vectors and gene therapy studies. ..
  16. Rozmus J, Junker A, Thibodeau M, Grenier D, Turvey S, Yacoub W, et al. Severe combined immunodeficiency (SCID) in Canadian children: a national surveillance study. J Clin Immunol. 2013;33:1310-6 pubmed
    ..The high mortality rate, due primarily to infection, suggests that early diagnosis by newborn screening followed by HSCT could significantly benefit children with SCID. ..
  17. Farazi T, Ten Hoeve J, Brown M, Mihailovic A, Horlings H, van de Vijver M, et al. Identification of distinct miRNA target regulation between breast cancer molecular subtypes using AGO2-PAR-CLIP and patient datasets. Genome Biol. 2014;15:R9 pubmed publisher
    ..We combined PAR-CLIP data with patient expression data to predict regulatory miRNAs, revealing potential therapeutic targets and prognostic markers in breast cancer. ..
  18. Corbeau P, Kraus G, Wong Staal F. Transduction of human macrophages using a stable HIV-1/HIV-2-derived gene delivery system. Gene Ther. 1998;5:99-104 pubmed
    ..The availability of a stable HIV-based gene delivery system for macrophages, a key target cell in HIV infection; is an important advance in gene therapy for AIDS. ..
  19. Orriols M, Gomez Puerto M, Ten Dijke P. BMP type II receptor as a therapeutic target in pulmonary arterial hypertension. Cell Mol Life Sci. 2017;74:2979-2995 pubmed publisher
    ..Different mutations in BMPR2 and environmental/genetic factors make PAH a heterogeneous disease and it is thus likely that the best approach will be patient-tailored therapies. ..
  20. Zarogoulidis P, Kontakiotis T, Zarogoulidis K. Inhaled gene therapy in lung cancer: "as for the future, our task is not to foresee it, but to enable it". Ther Deliv. 2012;3:919-21 pubmed
  21. Flotte T. Therapeutic Germ Line Alteration: Has CRISPR/Cas9 Technology Forced the Question?. Hum Gene Ther. 2015;26:245-6 pubmed publisher
  22. Gootwine E, Abu Siam M, Obolensky A, Rosov A, Honig H, Nitzan T, et al. Gene Augmentation Therapy for a Missense Substitution in the cGMP-Binding Domain of Ovine CNGA3 Gene Restores Vision in Day-Blind Sheep. Invest Ophthalmol Vis Sci. 2017;58:1577-1584 pubmed publisher
    ..This novel mutation provides a large-animal model that is valid for most human CNGA3 ACHM patients; the majority of them carry missense rather than premature-termination mutations. ..
  23. Strobel I, Berchtold S, Götze A, Schulze U, Schuler G, Steinkasserer A. Human dendritic cells transfected with either RNA or DNA encoding influenza matrix protein M1 differ in their ability to stimulate cytotoxic T lymphocytes. Gene Ther. 2000;7:2028-35 pubmed
    ..This RNA transfection technique consequently represents a very promising tool for future immunotherapy strategies. ..
  24. Maitrias P, Metzinger Le Meuth V, Nader J, Reix T, Caus T, Metzinger L. The Involvement of miRNA in Carotid-Related Stroke. Arterioscler Thromb Vasc Biol. 2017;37:1608-1617 pubmed publisher
    ..Lastly, we describe the miRNAs' potential role as a biomarker of stroke. ..
  25. Philippou A, Christopoulos P, Koutsilieris D. Clinical studies in humans targeting the various components of the IGF system show lack of efficacy in the treatment of cancer. Mutat Res Rev Mutat Res. 2017;772:105-122 pubmed publisher
  26. Gallardo Rincón D, Marquez J, Celis E. Cancer immunotherapy without frontiers: 2nd Annual Immuno-Oncology Meeting of the Centro de Investigación de Cancer en Sonora (CICS), Ciudad Obregón, Sonora México, Dec 2-4, 2016. Cancer Immunol Immunother. 2017;66:1243-1247 pubmed publisher
    ..This meeting in immuno-oncology brought together clinicians and scientists from United States, Canada, and México with the goal of breaking down international walls and establishing new collaborations. ..
  27. Gianferante D, Mirabello L, Savage S. Germline and somatic genetics of osteosarcoma - connecting aetiology, biology and therapy. Nat Rev Endocrinol. 2017;13:480-491 pubmed publisher
  28. Greener M. The good, the bad and the ugly red tape of biomedical research. How could regulators lower bureaucratic hurdles in clinical research without compromising the safety of patients?. EMBO Rep. 2009;10:17-20 pubmed publisher
  29. Fischer A. Severe combined immunodeficiencies (SCID). Clin Exp Immunol. 2000;122:143-9 pubmed
  30. Mulligan R. Development of gene transfer technology. Hum Gene Ther. 2014;25:995-1002 pubmed publisher
  31. Chauhan A, Zubair S, Nadeem A, Ansari S, Ansari M, Mohammad O. Escheriosome-mediated cytosolic delivery of PLK1-specific siRNA: potential in treatment of liver cancer in BALB/c mice. Nanomedicine (Lond). 2014;9:407-20 pubmed publisher
    ..This novel therapeutic strategy may be applicable to a broad range of cancers in patients with the obstinate form of the disease. ..
  32. Uthaman S, Maya S, Jayakumar R, Cho C, Park I. Carbohydrate-based nanogels as drug and gene delivery systems. J Nanosci Nanotechnol. 2014;14:694-704 pubmed
    ..This review summarizes the role of natural polymer-based nanogels, especially carbohydrate-based nanogels as drug and gene delivery systems. ..
  33. Duroux Richard I, Giovannangeli C, Apparailly F. CRISPR-Cas9: A revolution in genome editing in rheumatic diseases. Joint Bone Spine. 2017;84:1-4 pubmed publisher
  34. Engel B, Kohn D. Stem cell directed gene therapy. Front Biosci. 1999;4:e26-33 pubmed
    ..New vector delivery systems, such as lentiviral vectors, need to be developed to ensure efficient gene transfer and persistent transgene expression to provide life-long resistance to the cells targeted by HIV-1. ..
  35. Flight M. Trial watch: Clinical trial boost for lentiviral gene therapy. Nat Rev Drug Discov. 2013;12:654 pubmed publisher
  36. Gaspar H, Swift S, Thrasher A. "Special exemptions": should they be put on trial?. Mol Ther. 2013;21:261-2 pubmed publisher
  37. Yan F, Li X, Li N, Zhang R, Wang Q, Ru Y, et al. Immunoproapoptotic molecule scFv-Fdt-tBid modified mesenchymal stem cells for prostate cancer dual-targeted therapy. Cancer Lett. 2017;402:32-42 pubmed publisher
    ..Collectively, this study represented a novel immunoproapoptotic molecule scFv-Fdt-tBid for γ-SM-positive tumors and demonstrated the therapeutic efficiency and safety of scFv-Fdt-tBid-modified MSCs against prostate cancers. ..
  38. Eichler F, Duncan C, Musolino P, Orchard P, de Oliveira S, Thrasher A, et al. Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy. N Engl J Med. 2017;377:1630-1638 pubmed publisher
    ..Cerebral adrenoleukodystrophy is characterized by demyelination and neurodegeneration. Disease progression, which leads to loss of neurologic function and death, can be halted only with allogeneic hematopoietic stem-cell transplantation...
  39. Zhang J, Xie L, Xu D, Yue S, Li Y, Guo X, et al. Targeting expression of antimicrobial peptide CAMA-Syn by adenovirus vector in macrophages inhibits the growth of intracellular bacteria. Gene. 2017;630:59-67 pubmed publisher
  40. Li Y, Zhuo B, Yin Y, Han T, Li S, Li Z, et al. Anti-cancer effect of oncolytic adenovirus-armed shRNA targeting MYCN gene on doxorubicin-resistant neuroblastoma cells. Biochem Biophys Res Commun. 2017;491:134-139 pubmed publisher
    ..Thus, this synergistic effect of ZD55-shMYCN in combination with doxorubicin provides a novel therapy strategy for doxorubicin-resistant neuroblastoma, and is a promising approach for further clinical development. ..
  41. Maoz R, Garfinkel B, Soreq H. Alzheimer's Disease and ncRNAs. Adv Exp Med Biol. 2017;978:337-361 pubmed publisher
    ..Finally, we explore the potential promise of these findings for future therapeutic applications. ..
  42. Mehta K, Lee J, Taha A, Avgerinos E, Chaer R. Vascular applications of contrast-enhanced ultrasound imaging. J Vasc Surg. 2017;66:266-274 pubmed publisher
    ..Overall, with proper training and credentialing of technicians, the clinical implications are innumerable as microbubble technology is rapidly bursting onto the scene of cardiovascular medicine. ..
  43. Oliveira C, Ribeiro A, Veiga F, Silveira I. Recent Advances in Nucleic Acid-Based Delivery: From Bench to Clinical Trials in Genetic Diseases. J Biomed Nanotechnol. 2016;12:841-62 pubmed
  44. Marchio S, Sidman R, Arap W, Pasqualini R. Brain endothelial cell-targeted gene therapy of neurovascular disorders. EMBO Mol Med. 2016;8:592-4 pubmed publisher
  45. Weill J, Reynaud C. The ups and downs of negative (and positive) selection of B cells. J Clin Invest. 2015;125:3748-50 pubmed publisher
    ..These results suggest that, in a normal situation, mature naive B cells undergo a positive selection step driven by self-antigens, kept in control by Tregs. ..
  46. Kao R, Murakami S, Beirne O. The use of biologic mediators and tissue engineering in dentistry. Periodontol 2000. 2009;50:127-53 pubmed publisher
  47. Melchiorri D, Pani L, Gasparini P, Cossu G, Ancans J, Borg J, et al. Regulatory evaluation of Glybera in Europe - two committees, one mission. Nat Rev Drug Discov. 2013;12:719 pubmed publisher
  48. Komajda M, Ruschitzka F. The year in cardiology 2015: heart failure. Eur Heart J. 2016;37:437-41 pubmed publisher
  49. Huang Z, Kataoka M, Chen J, Wu G, Ding J, Nie M, et al. Cardiomyocyte-enriched protein CIP protects against pathophysiological stresses and regulates cardiac homeostasis. J Clin Invest. 2015;125:4122-34 pubmed publisher
    ..Our studies identify CIP as a key regulator of cardiomyopathy that has potential as a therapeutic target to attenuate heart failure progression. ..
  50. Saunders K, Wang L, Joyce M, Yang Z, Balazs A, Cheng C, et al. Broadly Neutralizing Human Immunodeficiency Virus Type 1 Antibody Gene Transfer Protects Nonhuman Primates from Mucosal Simian-Human Immunodeficiency Virus Infection. J Virol. 2015;89:8334-45 pubmed
    ..5 weeks after treatment. Gene transfer of an anti-HIV antibody can therefore protect against infection by viruses that cause AIDS in primates when the host immune responses are controlled. ..
  51. Plate K, Risau W. Angiogenesis in malignant gliomas. Glia. 1995;15:339-47 pubmed
  52. Zhang X, Zhang J, Ma Y, Pei X, Liu Q, Lu B, et al. A cell-based single-stranded DNA aptamer specifically targets gastric cancer. Int J Biochem Cell Biol. 2014;46:1-8 pubmed
    ..In conclusion, we obtained a novel aptamer specifically targeting gastric cancer,which is an effective tool for both gastric cancer diagnosis and drug delivery. ..
  53. Cora D, Re A, Caselle M, Bussolino F. MicroRNA-mediated regulatory circuits: outlook and perspectives. Phys Biol. 2017;14:045001 pubmed publisher
    ..Finally, we will consider possible future developments, in particular regarding microRNA-mediated circuits involving long non-coding RNAs and epigenetic regulators. ..