Gene therapy: charting a future course--summary of a National Institutes of Health Workshop, April 12, 2013.

Recently, the gene therapy field has begun to experience clinical successes in a number of different diseases using various approaches and vectors. The workshop Gene Therapy: Charting a Future Course, sponsored by the National Institutes of Health (NIH) Office of Biotechnology Activities, brought together early and mid-career researchers to discuss the key scientific challenges and opportunities, ethical and communication issues, and NIH and foundation resources available to facilitate further clinical advances.

[1]  M. Hauser,et al.  RNAi-mediated Gene Silencing of Mutant Myotilin Improves Myopathy in LGMD1A Mice , 2014, Molecular therapy. Nucleic acids.

[2]  W. Hauswirth,et al.  Dual adeno-associated virus vectors result in efficient in vitro and in vivo expression of an oversized gene, MYO7A. , 2014, Human gene therapy methods.

[3]  Qing He,et al.  Efficacy and Toxicity Management of 19-28z CAR T Cell Therapy in B Cell Acute Lymphoblastic Leukemia , 2014, Science Translational Medicine.

[4]  U. Gerdemann,et al.  Safety and clinical efficacy of rapidly-generated trivirus-directed T cells as treatment for adenovirus, EBV, and CMV infections after allogeneic hematopoietic stem cell transplant. , 2013, Molecular therapy : the journal of the American Society of Gene Therapy.

[5]  R. Samulski,et al.  An emerging adeno-associated viral vector pipeline for cardiac gene therapy. , 2013, Human gene therapy.

[6]  R. Crystal,et al.  Gene therapy to stimulate angiogenesis to treat diffuse coronary artery disease. , 2013, Human gene therapy.

[7]  D. Elashoff,et al.  Modification of hematopoietic stem/progenitor cells with CD19-specific chimeric antigen receptors as a novel approach for cancer immunotherapy. , 2013, Human gene therapy.

[8]  Y. Kew,et al.  Combinational Targeting Offsets Antigen Escape and Enhances Effector Functions of Adoptively Transferred T Cells in Glioblastoma , 2013, Molecular therapy : the journal of the American Society of Gene Therapy.

[9]  A. Thrasher,et al.  Gene therapy for PIDs: Progress, pitfalls and prospects , 2013, Gene.

[10]  A. Fischer,et al.  Gene therapy of primary T cell immunodeficiencies. , 2013, Gene.

[11]  Michael L Kaufman,et al.  β-globin gene transfer to human bone marrow for sickle cell disease. , 2013, The Journal of clinical investigation.

[12]  Matthew L Baker,et al.  TanCAR: A Novel Bispecific Chimeric Antigen Receptor for Cancer Immunotherapy , 2013, Molecular therapy. Nucleic acids.

[13]  Michael L Kaufman,et al.  Allelic exclusion and peripheral reconstitution by TCR transgenic T cells arising from transduced human hematopoietic stem/progenitor cells. , 2013, Molecular therapy : the journal of the American Society of Gene Therapy.

[14]  Bernd Hauck,et al.  Chimeric antigen receptor-modified T cells for acute lymphoid leukemia. , 2013, The New England journal of medicine.

[15]  W. Hauswirth,et al.  AAV-mediated gene therapy in the guanylate cyclase (RetGC1/RetGC2) double knockout mouse model of Leber congenital amaurosis. , 2013, Human gene therapy.

[16]  W. Hauswirth,et al.  Retinal gene therapy with a large MYO7A cDNA using adeno-associated virus , 2013, Gene Therapy.

[17]  Aaron R Cooper,et al.  Gene therapy for adenosine deaminase-deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans. , 2012, Blood.

[18]  K. Purpura,et al.  Long-term expression and safety of administration of AAVrh.10hCLN2 to the brain of rats and nonhuman primates for the treatment of late infantile neuronal ceroid lipofuscinosis. , 2012, Human gene therapy methods.

[19]  Paul D. Gamlin,et al.  The human rhodopsin kinase promoter in an AAV5 vector confers rod- and cone-specific expression in the primate retina. , 2012, Human gene therapy.

[20]  Lucas V. Greder,et al.  Transgene‐Free Disease‐Specific Induced Pluripotent Stem Cells from Patients with Type 1 and Type 2 Diabetes , 2012, Stem cells translational medicine.

[21]  Jinjuan Wang,et al.  CD20-specific adoptive immunotherapy for lymphoma using a chimeric antigen receptor with both CD28 and 4-1BB domains: pilot clinical trial results. , 2012, Blood.

[22]  David V Schaffer,et al.  The AAV Vector Toolkit: Poised at the Clinical Crossroads. , 2012, Molecular therapy : the journal of the American Society of Gene Therapy.

[23]  G. Dranoff,et al.  Combining immunotherapy and targeted therapies in cancer treatment , 2012, Nature Reviews Cancer.

[24]  P. Davis An unparalleled engine for discovery and clinical introduction: the clinical and translational science awards and gene therapy. , 2012, Human gene therapy.

[25]  M. Chuah,et al.  Clinical progress in gene therapy: sustained partial correction of the bleeding disorder in patients suffering from severe hemophilia B. , 2012, Human gene therapy.

[26]  Pratima Chowdary,et al.  Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. , 2011, The New England journal of medicine.

[27]  A. Thrasher,et al.  Gene therapy for primary immunodeficiency , 2011, Current opinion in pediatrics.

[28]  Adrian P Gee,et al.  Inducible apoptosis as a safety switch for adoptive cell therapy. , 2011, The New England journal of medicine.

[29]  S. Frantz Engineered T-cell therapy shows efficacy in blood cancer , 2011, Nature Biotechnology.

[30]  W. Hauswirth,et al.  Long-term preservation of cone photoreceptors and restoration of cone function by gene therapy in the guanylate cyclase-1 knockout (GC1KO) mouse. , 2011, Investigative ophthalmology & visual science.

[31]  A. Bagg,et al.  Chimeric antigen receptor-modified T cells in chronic lymphoid leukemia. , 2011, The New England journal of medicine.

[32]  Hao Liu,et al.  CD28 costimulation improves expansion and persistence of chimeric antigen receptor-modified T cells in lymphoma patients. , 2011, The Journal of clinical investigation.

[33]  Frederic D. Bushman,et al.  Efficacy of gene therapy for X-linked severe combined immunodeficiency. , 2010, The New England journal of medicine.

[34]  W. Hauswirth,et al.  Functional and Behavioral Restoration of Vision by Gene Therapy in the Guanylate Cyclase-1 (GC1) Knockout Mouse , 2010, PloS one.

[35]  Alessandro Aiuti,et al.  Gene therapy for immunodeficiency due to adenosine deaminase deficiency. , 2009, The New England journal of medicine.

[36]  I. Kerridge,et al.  Accepting risk in clinical research: is the gene therapy field becoming too risk-averse? , 2009, Molecular therapy : the journal of the American Society of Gene Therapy.

[37]  J. Kimmelman Tomorrow, Interrupted?: Risk, Ethics, and Medical Advance in Gene Transfer. , 2009, Molecular therapy : the journal of the American Society of Gene Therapy.

[38]  A. Zwinderman,et al.  Intramuscular administration of AAV1-lipoprotein lipase S447X lowers triglycerides in lipoprotein lipase-deficient patients. , 2008, Arteriosclerosis, thrombosis, and vascular biology.

[39]  Hao Liu,et al.  Virus-specific T cells engineered to coexpress tumor-specific receptors: persistence and antitumor activity in individuals with neuroblastoma , 2008, Nature Medicine.

[40]  W. Hauswirth,et al.  Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial. , 2008, Human gene therapy.

[41]  Kathleen A. Marshall,et al.  Safety and efficacy of gene transfer for Leber's congenital amaurosis. , 2008, The New England journal of medicine.

[42]  D. Zallen US gene therapy in crisis. , 2000 .

[43]  Z. Eshhar,et al.  Specific activation and targeting of cytotoxic lymphocytes through chimeric single chains consisting of antibody-binding domains and the gamma or zeta subunits of the immunoglobulin and T-cell receptors. , 1993, Proceedings of the National Academy of Sciences of the United States of America.

[44]  A. J. Roman,et al.  Determining consequences of retinal membrane guanylyl cyclase (RetGC1) deficiency in human Leber congenital amaurosis en route to therapy: residual cone-photoreceptor vision correlates with biochemical properties of the mutants. , 2013, Human molecular genetics.

[45]  A. Terzic,et al.  Intrapatient variations in type 1 diabetes-specific iPS cell differentiation into insulin-producing cells. , 2013, Molecular therapy : the journal of the American Society of Gene Therapy.

[46]  D. Maloney,et al.  CD 20-specific adoptive immunotherapy for lymphoma using a chimeric antigen receptor with both CD 28 and 4-1 BB domains : pilot clinical trial results , 2012 .

[47]  A. Lawson,et al.  Artificial T-cell receptors. , 2003, Cytotherapy.

[48]  D Teichler Zallen,et al.  US gene therapy in crisis. , 2000, Trends in genetics : TIG.