ArticleThe New England journal of medicine2023
Death after High-Dose rAAV9 Gene Therapy in a Patient with Duchenne's Muscular Dystrophy.
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In one paragraph
Article in The New England journal of medicine, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 187 papers, 1 of them a synthesis that pooled it.
0numbers the graph read from it
0cells of the map it votes in
187citing papers in PubMed, 1 pooled it
43.6field-weighted citation impact, top 1% of its field
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
187 citing papers in PubMed, 1 synthesis or guideline pooled it, 283 citations in OpenAlex.
- Incidence, timing, and clinical significance of adverse immune events after gene replacement therapy: A systematic review and meta-analysis.Molecular therapy : the journal of the American Society of Gene Therapy · 2026Pooled it
- AAV9 Gene Therapy in Type II GM1 Gangliosidosis - A Phase 1-2 Trial.The New England journal of medicine · 2026Trial
- Sequence-based artificial intelligence-guided inhibitory peptides to counteract AAV neutralizing antibodies in gene therapy.Molecular therapy. Advances · 2026Article
- Global epidemiology of myocarditis: disparities in age, sex and geography.Nature reviews. Cardiology · 2026Review
- Reduced Cx43 S-nitrosylation protects against cardiac stress-induced arrhythmias and myocardial injury in Duchenne muscular dystrophy.Cardiovascular research · 2026Article
- The global patent landscape of CRISPR-Cas applications in medicine.Nature biotechnology · 2026Article
- Therapeutic upregulation of gene expression in inherited cardiomyopathies from current approaches to future directions.Nature cardiovascular research · 2026Review
- Patient-derived teratomas as a humanized platform for dystrophin restoration by ex vivo cell therapy and in vivo base editing.Experimental & molecular medicine · 2026Article
- Expanded Access use of Intravenous Gene Transfer with AAV9-GLB1 in a Juvenile GM1 Gangliosidosis Participant.medRxiv : the preprint server for health sciences · 2026Article
- Article
- Decoding Nonlinearities in AAV-Based Gene Therapy Using PBPK Modelling.The AAPS journal · 2026Article
- Immune mechanisms and pathophysiology of T cell-mediated pediatric acute liver failure (TC-PALF).Hepatology communications · 2026Review
- Combination S100A1 and ARC gene therapy as a treatment for Duchenne muscular dystrophy cardiomyopathy.JCI insight · 2026Article
- Death following high-dose AAV9 gene therapy in a patient with advanced SMA-PME.Molecular therapy : the journal of the American Society of Gene Therapy · 2026Article
- AAV9-mediated GAA gene therapy following enzyme replacement therapy discontinuation in children with infantile-onset Pompe disease.EClinicalMedicine · 2026Article
- Structural basis of liver de-targeting and neuronal tropism of CNS-targeted AAV capsids.Molecular therapy : the journal of the American Society of Gene Therapy · 2026Article
- Attenuating AAV-triggered innate immunity in the adult mouse nervous systemActa pharmaceutica Sinica. B · 2026Article
- Decoding PANoptosis: Crosstalk of cell death pathways in immunity and inflammation.Immunologic research · 2026Review
- Skeletal-muscle-targeted non-viral delivery of full-length DMD mRNA for Duchenne muscular dystrophy.Nature biomedical engineering · 2026Article
- Engineering novel AAV capsids by broadly attenuated and subsequent muscle-specific tropism in mice and NHPs.Molecular therapy. Advances · 2026Article
127 more citing papers are in PubMed but not listed here.
Corrections and comments
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Authors and funding
25 authors at 5 institutions in 2 countries.
Angela LekFrom the Department of Genetics, Yale School of Medicine, New Haven (A.L., K.M., S.H., K.W., S. Pajusalu, M.L.), and Cure Rare Disease, Woodbridge (R.H.) - both in Connecticut; the Departments of Pediatrics (B.W., A.K., R.A., D.K., T.F.) and Neurology (A.R.B.) and Horae Gene Therapy Center and the Li Weibo Institute for Rare Diseases Research (A.K., M.B., K. Sylvia, A.R.B., R.A., D.K., S. Parajuli, T.G., T.F.), University of Massachusetts Chan Medical School, Worcester, the Department of Pathology (J.P., C.K.C., H.L.), the Division of Genetics (J.M.S.), and Department of Cardiology (J.L.), Boston Children's Hospital, and Harvard Medical School (J.P., C.K.C., H.L.), Boston, and Charles River Laboratories, Wilmington (L.B., K. Sutton) - all in Massachusetts; the Department of Clinical Genetics, Institute of Clinical Medicine, University of Tartu (S. Pajusalu), and the Genetics and Personalized Medicine Clinic, Tartu University Hospital (S. Pajusalu) - both in Tartu, Estonia; and Regulatory Innovation, Raleigh, NC (D.B.).ORCID 0000-0001-5889-2169
Brenda WongFrom the Department of Genetics, Yale School of Medicine, New Haven (A.L., K.M., S.H., K.W., S. Pajusalu, M.L.), and Cure Rare Disease, Woodbridge (R.H.) - both in Connecticut; the Departments of Pediatrics (B.W., A.K., R.A., D.K., T.F.) and Neurology (A.R.B.) and Horae Gene Therapy Center and the Li Weibo Institute for Rare Diseases Research (A.K., M.B., K. Sylvia, A.R.B., R.A., D.K., S. Parajuli, T.G., T.F.), University of Massachusetts Chan Medical School, Worcester, the Department of Pathology (J.P., C.K.C., H.L.), the Division of Genetics (J.M.S.), and Department of Cardiology (J.L.), Boston Children's Hospital, and Harvard Medical School (J.P., C.K.C., H.L.), Boston, and Charles River Laboratories, Wilmington (L.B., K. Sutton) - all in Massachusetts; the Department of Clinical Genetics, Institute of Clinical Medicine, University of Tartu (S. Pajusalu), and the Genetics and Personalized Medicine Clinic, Tartu University Hospital (S. Pajusalu) - both in Tartu, Estonia; and Regulatory Innovation, Raleigh, NC (D.B.).
Allison KeelerFrom the Department of Genetics, Yale School of Medicine, New Haven (A.L., K.M., S.H., K.W., S. Pajusalu, M.L.), and Cure Rare Disease, Woodbridge (R.H.) - both in Connecticut; the Departments of Pediatrics (B.W., A.K., R.A., D.K., T.F.) and Neurology (A.R.B.) and Horae Gene Therapy Center and the Li Weibo Institute for Rare Diseases Research (A.K., M.B., K. Sylvia, A.R.B., R.A., D.K., S. Parajuli, T.G., T.F.), University of Massachusetts Chan Medical School, Worcester, the Department of Pathology (J.P., C.K.C., H.L.), the Division of Genetics (J.M.S.), and Department of Cardiology (J.L.), Boston Children's Hospital, and Harvard Medical School (J.P., C.K.C., H.L.), Boston, and Charles River Laboratories, Wilmington (L.B., K. Sutton) - all in Massachusetts; the Department of Clinical Genetics, Institute of Clinical Medicine, University of Tartu (S. Pajusalu), and the Genetics and Personalized Medicine Clinic, Tartu University Hospital (S. Pajusalu) - both in Tartu, Estonia; and Regulatory Innovation, Raleigh, NC (D.B.).
Meghan BlackwoodFrom the Department of Genetics, Yale School of Medicine, New Haven (A.L., K.M., S.H., K.W., S. Pajusalu, M.L.), and Cure Rare Disease, Woodbridge (R.H.) - both in Connecticut; the Departments of Pediatrics (B.W., A.K., R.A., D.K., T.F.) and Neurology (A.R.B.) and Horae Gene Therapy Center and the Li Weibo Institute for Rare Diseases Research (A.K., M.B., K. Sylvia, A.R.B., R.A., D.K., S. Parajuli, T.G., T.F.), University of Massachusetts Chan Medical School, Worcester, the Department of Pathology (J.P., C.K.C., H.L.), the Division of Genetics (J.M.S.), and Department of Cardiology (J.L.), Boston Children's Hospital, and Harvard Medical School (J.P., C.K.C., H.L.), Boston, and Charles River Laboratories, Wilmington (L.B., K. Sutton) - all in Massachusetts; the Department of Clinical Genetics, Institute of Clinical Medicine, University of Tartu (S. Pajusalu), and the Genetics and Personalized Medicine Clinic, Tartu University Hospital (S. Pajusalu) - both in Tartu, Estonia; and Regulatory Innovation, Raleigh, NC (D.B.).ORCID 0000-0002-3051-6548
Kaiyue MaFrom the Department of Genetics, Yale School of Medicine, New Haven (A.L., K.M., S.H., K.W., S. Pajusalu, M.L.), and Cure Rare Disease, Woodbridge (R.H.) - both in Connecticut; the Departments of Pediatrics (B.W., A.K., R.A., D.K., T.F.) and Neurology (A.R.B.) and Horae Gene Therapy Center and the Li Weibo Institute for Rare Diseases Research (A.K., M.B., K. Sylvia, A.R.B., R.A., D.K., S. Parajuli, T.G., T.F.), University of Massachusetts Chan Medical School, Worcester, the Department of Pathology (J.P., C.K.C., H.L.), the Division of Genetics (J.M.S.), and Department of Cardiology (J.L.), Boston Children's Hospital, and Harvard Medical School (J.P., C.K.C., H.L.), Boston, and Charles River Laboratories, Wilmington (L.B., K. Sutton) - all in Massachusetts; the Department of Clinical Genetics, Institute of Clinical Medicine, University of Tartu (S. Pajusalu), and the Genetics and Personalized Medicine Clinic, Tartu University Hospital (S. Pajusalu) - both in Tartu, Estonia; and Regulatory Innovation, Raleigh, NC (D.B.).ORCID 0009-0001-4302-3371
Shushu HuangFrom the Department of Genetics, Yale School of Medicine, New Haven (A.L., K.M., S.H., K.W., S. Pajusalu, M.L.), and Cure Rare Disease, Woodbridge (R.H.) - both in Connecticut; the Departments of Pediatrics (B.W., A.K., R.A., D.K., T.F.) and Neurology (A.R.B.) and Horae Gene Therapy Center and the Li Weibo Institute for Rare Diseases Research (A.K., M.B., K. Sylvia, A.R.B., R.A., D.K., S. Parajuli, T.G., T.F.), University of Massachusetts Chan Medical School, Worcester, the Department of Pathology (J.P., C.K.C., H.L.), the Division of Genetics (J.M.S.), and Department of Cardiology (J.L.), Boston Children's Hospital, and Harvard Medical School (J.P., C.K.C., H.L.), Boston, and Charles River Laboratories, Wilmington (L.B., K. Sutton) - all in Massachusetts; the Department of Clinical Genetics, Institute of Clinical Medicine, University of Tartu (S. Pajusalu), and the Genetics and Personalized Medicine Clinic, Tartu University Hospital (S. Pajusalu) - both in Tartu, Estonia; and Regulatory Innovation, Raleigh, NC (D.B.).ORCID 0000-0002-0220-4395
Katelyn SylviaFrom the Department of Genetics, Yale School of Medicine, New Haven (A.L., K.M., S.H., K.W., S. Pajusalu, M.L.), and Cure Rare Disease, Woodbridge (R.H.) - both in Connecticut; the Departments of Pediatrics (B.W., A.K., R.A., D.K., T.F.) and Neurology (A.R.B.) and Horae Gene Therapy Center and the Li Weibo Institute for Rare Diseases Research (A.K., M.B., K. Sylvia, A.R.B., R.A., D.K., S. Parajuli, T.G., T.F.), University of Massachusetts Chan Medical School, Worcester, the Department of Pathology (J.P., C.K.C., H.L.), the Division of Genetics (J.M.S.), and Department of Cardiology (J.L.), Boston Children's Hospital, and Harvard Medical School (J.P., C.K.C., H.L.), Boston, and Charles River Laboratories, Wilmington (L.B., K. Sutton) - all in Massachusetts; the Department of Clinical Genetics, Institute of Clinical Medicine, University of Tartu (S. Pajusalu), and the Genetics and Personalized Medicine Clinic, Tartu University Hospital (S. Pajusalu) - both in Tartu, Estonia; and Regulatory Innovation, Raleigh, NC (D.B.).
A Rita BatistaFrom the Department of Genetics, Yale School of Medicine, New Haven (A.L., K.M., S.H., K.W., S. Pajusalu, M.L.), and Cure Rare Disease, Woodbridge (R.H.) - both in Connecticut; the Departments of Pediatrics (B.W., A.K., R.A., D.K., T.F.) and Neurology (A.R.B.) and Horae Gene Therapy Center and the Li Weibo Institute for Rare Diseases Research (A.K., M.B., K. Sylvia, A.R.B., R.A., D.K., S. Parajuli, T.G., T.F.), University of Massachusetts Chan Medical School, Worcester, the Department of Pathology (J.P., C.K.C., H.L.), the Division of Genetics (J.M.S.), and Department of Cardiology (J.L.), Boston Children's Hospital, and Harvard Medical School (J.P., C.K.C., H.L.), Boston, and Charles River Laboratories, Wilmington (L.B., K. Sutton) - all in Massachusetts; the Department of Clinical Genetics, Institute of Clinical Medicine, University of Tartu (S. Pajusalu), and the Genetics and Personalized Medicine Clinic, Tartu University Hospital (S. Pajusalu) - both in Tartu, Estonia; and Regulatory Innovation, Raleigh, NC (D.B.).
Rebecca ArtinianFrom the Department of Genetics, Yale School of Medicine, New Haven (A.L., K.M., S.H., K.W., S. Pajusalu, M.L.), and Cure Rare Disease, Woodbridge (R.H.) - both in Connecticut; the Departments of Pediatrics (B.W., A.K., R.A., D.K., T.F.) and Neurology (A.R.B.) and Horae Gene Therapy Center and the Li Weibo Institute for Rare Diseases Research (A.K., M.B., K. Sylvia, A.R.B., R.A., D.K., S. Parajuli, T.G., T.F.), University of Massachusetts Chan Medical School, Worcester, the Department of Pathology (J.P., C.K.C., H.L.), the Division of Genetics (J.M.S.), and Department of Cardiology (J.L.), Boston Children's Hospital, and Harvard Medical School (J.P., C.K.C., H.L.), Boston, and Charles River Laboratories, Wilmington (L.B., K. Sutton) - all in Massachusetts; the Department of Clinical Genetics, Institute of Clinical Medicine, University of Tartu (S. Pajusalu), and the Genetics and Personalized Medicine Clinic, Tartu University Hospital (S. Pajusalu) - both in Tartu, Estonia; and Regulatory Innovation, Raleigh, NC (D.B.).
Danielle KokoskiFrom the Department of Genetics, Yale School of Medicine, New Haven (A.L., K.M., S.H., K.W., S. Pajusalu, M.L.), and Cure Rare Disease, Woodbridge (R.H.) - both in Connecticut; the Departments of Pediatrics (B.W., A.K., R.A., D.K., T.F.) and Neurology (A.R.B.) and Horae Gene Therapy Center and the Li Weibo Institute for Rare Diseases Research (A.K., M.B., K. Sylvia, A.R.B., R.A., D.K., S. Parajuli, T.G., T.F.), University of Massachusetts Chan Medical School, Worcester, the Department of Pathology (J.P., C.K.C., H.L.), the Division of Genetics (J.M.S.), and Department of Cardiology (J.L.), Boston Children's Hospital, and Harvard Medical School (J.P., C.K.C., H.L.), Boston, and Charles River Laboratories, Wilmington (L.B., K. Sutton) - all in Massachusetts; the Department of Clinical Genetics, Institute of Clinical Medicine, University of Tartu (S. Pajusalu), and the Genetics and Personalized Medicine Clinic, Tartu University Hospital (S. Pajusalu) - both in Tartu, Estonia; and Regulatory Innovation, Raleigh, NC (D.B.).
Shestruma ParajuliFrom the Department of Genetics, Yale School of Medicine, New Haven (A.L., K.M., S.H., K.W., S. Pajusalu, M.L.), and Cure Rare Disease, Woodbridge (R.H.) - both in Connecticut; the Departments of Pediatrics (B.W., A.K., R.A., D.K., T.F.) and Neurology (A.R.B.) and Horae Gene Therapy Center and the Li Weibo Institute for Rare Diseases Research (A.K., M.B., K. Sylvia, A.R.B., R.A., D.K., S. Parajuli, T.G., T.F.), University of Massachusetts Chan Medical School, Worcester, the Department of Pathology (J.P., C.K.C., H.L.), the Division of Genetics (J.M.S.), and Department of Cardiology (J.L.), Boston Children's Hospital, and Harvard Medical School (J.P., C.K.C., H.L.), Boston, and Charles River Laboratories, Wilmington (L.B., K. Sutton) - all in Massachusetts; the Department of Clinical Genetics, Institute of Clinical Medicine, University of Tartu (S. Pajusalu), and the Genetics and Personalized Medicine Clinic, Tartu University Hospital (S. Pajusalu) - both in Tartu, Estonia; and Regulatory Innovation, Raleigh, NC (D.B.).
Juan PutraFrom the Department of Genetics, Yale School of Medicine, New Haven (A.L., K.M., S.H., K.W., S. Pajusalu, M.L.), and Cure Rare Disease, Woodbridge (R.H.) - both in Connecticut; the Departments of Pediatrics (B.W., A.K., R.A., D.K., T.F.) and Neurology (A.R.B.) and Horae Gene Therapy Center and the Li Weibo Institute for Rare Diseases Research (A.K., M.B., K. Sylvia, A.R.B., R.A., D.K., S. Parajuli, T.G., T.F.), University of Massachusetts Chan Medical School, Worcester, the Department of Pathology (J.P., C.K.C., H.L.), the Division of Genetics (J.M.S.), and Department of Cardiology (J.L.), Boston Children's Hospital, and Harvard Medical School (J.P., C.K.C., H.L.), Boston, and Charles River Laboratories, Wilmington (L.B., K. Sutton) - all in Massachusetts; the Department of Clinical Genetics, Institute of Clinical Medicine, University of Tartu (S. Pajusalu), and the Genetics and Personalized Medicine Clinic, Tartu University Hospital (S. Pajusalu) - both in Tartu, Estonia; and Regulatory Innovation, Raleigh, NC (D.B.).
C Katte CarreonFrom the Department of Genetics, Yale School of Medicine, New Haven (A.L., K.M., S.H., K.W., S. Pajusalu, M.L.), and Cure Rare Disease, Woodbridge (R.H.) - both in Connecticut; the Departments of Pediatrics (B.W., A.K., R.A., D.K., T.F.) and Neurology (A.R.B.) and Horae Gene Therapy Center and the Li Weibo Institute for Rare Diseases Research (A.K., M.B., K. Sylvia, A.R.B., R.A., D.K., S. Parajuli, T.G., T.F.), University of Massachusetts Chan Medical School, Worcester, the Department of Pathology (J.P., C.K.C., H.L.), the Division of Genetics (J.M.S.), and Department of Cardiology (J.L.), Boston Children's Hospital, and Harvard Medical School (J.P., C.K.C., H.L.), Boston, and Charles River Laboratories, Wilmington (L.B., K. Sutton) - all in Massachusetts; the Department of Clinical Genetics, Institute of Clinical Medicine, University of Tartu (S. Pajusalu), and the Genetics and Personalized Medicine Clinic, Tartu University Hospital (S. Pajusalu) - both in Tartu, Estonia; and Regulatory Innovation, Raleigh, NC (D.B.).
Hart LidovFrom the Department of Genetics, Yale School of Medicine, New Haven (A.L., K.M., S.H., K.W., S. Pajusalu, M.L.), and Cure Rare Disease, Woodbridge (R.H.) - both in Connecticut; the Departments of Pediatrics (B.W., A.K., R.A., D.K., T.F.) and Neurology (A.R.B.) and Horae Gene Therapy Center and the Li Weibo Institute for Rare Diseases Research (A.K., M.B., K. Sylvia, A.R.B., R.A., D.K., S. Parajuli, T.G., T.F.), University of Massachusetts Chan Medical School, Worcester, the Department of Pathology (J.P., C.K.C., H.L.), the Division of Genetics (J.M.S.), and Department of Cardiology (J.L.), Boston Children's Hospital, and Harvard Medical School (J.P., C.K.C., H.L.), Boston, and Charles River Laboratories, Wilmington (L.B., K. Sutton) - all in Massachusetts; the Department of Clinical Genetics, Institute of Clinical Medicine, University of Tartu (S. Pajusalu), and the Genetics and Personalized Medicine Clinic, Tartu University Hospital (S. Pajusalu) - both in Tartu, Estonia; and Regulatory Innovation, Raleigh, NC (D.B.).
Keryn WoodmanFrom the Department of Genetics, Yale School of Medicine, New Haven (A.L., K.M., S.H., K.W., S. Pajusalu, M.L.), and Cure Rare Disease, Woodbridge (R.H.) - both in Connecticut; the Departments of Pediatrics (B.W., A.K., R.A., D.K., T.F.) and Neurology (A.R.B.) and Horae Gene Therapy Center and the Li Weibo Institute for Rare Diseases Research (A.K., M.B., K. Sylvia, A.R.B., R.A., D.K., S. Parajuli, T.G., T.F.), University of Massachusetts Chan Medical School, Worcester, the Department of Pathology (J.P., C.K.C., H.L.), the Division of Genetics (J.M.S.), and Department of Cardiology (J.L.), Boston Children's Hospital, and Harvard Medical School (J.P., C.K.C., H.L.), Boston, and Charles River Laboratories, Wilmington (L.B., K. Sutton) - all in Massachusetts; the Department of Clinical Genetics, Institute of Clinical Medicine, University of Tartu (S. Pajusalu), and the Genetics and Personalized Medicine Clinic, Tartu University Hospital (S. Pajusalu) - both in Tartu, Estonia; and Regulatory Innovation, Raleigh, NC (D.B.).
Sander PajusaluFrom the Department of Genetics, Yale School of Medicine, New Haven (A.L., K.M., S.H., K.W., S. Pajusalu, M.L.), and Cure Rare Disease, Woodbridge (R.H.) - both in Connecticut; the Departments of Pediatrics (B.W., A.K., R.A., D.K., T.F.) and Neurology (A.R.B.) and Horae Gene Therapy Center and the Li Weibo Institute for Rare Diseases Research (A.K., M.B., K. Sylvia, A.R.B., R.A., D.K., S. Parajuli, T.G., T.F.), University of Massachusetts Chan Medical School, Worcester, the Department of Pathology (J.P., C.K.C., H.L.), the Division of Genetics (J.M.S.), and Department of Cardiology (J.L.), Boston Children's Hospital, and Harvard Medical School (J.P., C.K.C., H.L.), Boston, and Charles River Laboratories, Wilmington (L.B., K. Sutton) - all in Massachusetts; the Department of Clinical Genetics, Institute of Clinical Medicine, University of Tartu (S. Pajusalu), and the Genetics and Personalized Medicine Clinic, Tartu University Hospital (S. Pajusalu) - both in Tartu, Estonia; and Regulatory Innovation, Raleigh, NC (D.B.).
Janelle M SpinazzolaFrom the Department of Genetics, Yale School of Medicine, New Haven (A.L., K.M., S.H., K.W., S. Pajusalu, M.L.), and Cure Rare Disease, Woodbridge (R.H.) - both in Connecticut; the Departments of Pediatrics (B.W., A.K., R.A., D.K., T.F.) and Neurology (A.R.B.) and Horae Gene Therapy Center and the Li Weibo Institute for Rare Diseases Research (A.K., M.B., K. Sylvia, A.R.B., R.A., D.K., S. Parajuli, T.G., T.F.), University of Massachusetts Chan Medical School, Worcester, the Department of Pathology (J.P., C.K.C., H.L.), the Division of Genetics (J.M.S.), and Department of Cardiology (J.L.), Boston Children's Hospital, and Harvard Medical School (J.P., C.K.C., H.L.), Boston, and Charles River Laboratories, Wilmington (L.B., K. Sutton) - all in Massachusetts; the Department of Clinical Genetics, Institute of Clinical Medicine, University of Tartu (S. Pajusalu), and the Genetics and Personalized Medicine Clinic, Tartu University Hospital (S. Pajusalu) - both in Tartu, Estonia; and Regulatory Innovation, Raleigh, NC (D.B.).ORCID 0000-0002-7141-5487
Thomas GallagherFrom the Department of Genetics, Yale School of Medicine, New Haven (A.L., K.M., S.H., K.W., S. Pajusalu, M.L.), and Cure Rare Disease, Woodbridge (R.H.) - both in Connecticut; the Departments of Pediatrics (B.W., A.K., R.A., D.K., T.F.) and Neurology (A.R.B.) and Horae Gene Therapy Center and the Li Weibo Institute for Rare Diseases Research (A.K., M.B., K. Sylvia, A.R.B., R.A., D.K., S. Parajuli, T.G., T.F.), University of Massachusetts Chan Medical School, Worcester, the Department of Pathology (J.P., C.K.C., H.L.), the Division of Genetics (J.M.S.), and Department of Cardiology (J.L.), Boston Children's Hospital, and Harvard Medical School (J.P., C.K.C., H.L.), Boston, and Charles River Laboratories, Wilmington (L.B., K. Sutton) - all in Massachusetts; the Department of Clinical Genetics, Institute of Clinical Medicine, University of Tartu (S. Pajusalu), and the Genetics and Personalized Medicine Clinic, Tartu University Hospital (S. Pajusalu) - both in Tartu, Estonia; and Regulatory Innovation, Raleigh, NC (D.B.).
Joan LaRovereFrom the Department of Genetics, Yale School of Medicine, New Haven (A.L., K.M., S.H., K.W., S. Pajusalu, M.L.), and Cure Rare Disease, Woodbridge (R.H.) - both in Connecticut; the Departments of Pediatrics (B.W., A.K., R.A., D.K., T.F.) and Neurology (A.R.B.) and Horae Gene Therapy Center and the Li Weibo Institute for Rare Diseases Research (A.K., M.B., K. Sylvia, A.R.B., R.A., D.K., S. Parajuli, T.G., T.F.), University of Massachusetts Chan Medical School, Worcester, the Department of Pathology (J.P., C.K.C., H.L.), the Division of Genetics (J.M.S.), and Department of Cardiology (J.L.), Boston Children's Hospital, and Harvard Medical School (J.P., C.K.C., H.L.), Boston, and Charles River Laboratories, Wilmington (L.B., K. Sutton) - all in Massachusetts; the Department of Clinical Genetics, Institute of Clinical Medicine, University of Tartu (S. Pajusalu), and the Genetics and Personalized Medicine Clinic, Tartu University Hospital (S. Pajusalu) - both in Tartu, Estonia; and Regulatory Innovation, Raleigh, NC (D.B.).
Diane BaldersonFrom the Department of Genetics, Yale School of Medicine, New Haven (A.L., K.M., S.H., K.W., S. Pajusalu, M.L.), and Cure Rare Disease, Woodbridge (R.H.) - both in Connecticut; the Departments of Pediatrics (B.W., A.K., R.A., D.K., T.F.) and Neurology (A.R.B.) and Horae Gene Therapy Center and the Li Weibo Institute for Rare Diseases Research (A.K., M.B., K. Sylvia, A.R.B., R.A., D.K., S. Parajuli, T.G., T.F.), University of Massachusetts Chan Medical School, Worcester, the Department of Pathology (J.P., C.K.C., H.L.), the Division of Genetics (J.M.S.), and Department of Cardiology (J.L.), Boston Children's Hospital, and Harvard Medical School (J.P., C.K.C., H.L.), Boston, and Charles River Laboratories, Wilmington (L.B., K. Sutton) - all in Massachusetts; the Department of Clinical Genetics, Institute of Clinical Medicine, University of Tartu (S. Pajusalu), and the Genetics and Personalized Medicine Clinic, Tartu University Hospital (S. Pajusalu) - both in Tartu, Estonia; and Regulatory Innovation, Raleigh, NC (D.B.).
Lauren BlackFrom the Department of Genetics, Yale School of Medicine, New Haven (A.L., K.M., S.H., K.W., S. Pajusalu, M.L.), and Cure Rare Disease, Woodbridge (R.H.) - both in Connecticut; the Departments of Pediatrics (B.W., A.K., R.A., D.K., T.F.) and Neurology (A.R.B.) and Horae Gene Therapy Center and the Li Weibo Institute for Rare Diseases Research (A.K., M.B., K. Sylvia, A.R.B., R.A., D.K., S. Parajuli, T.G., T.F.), University of Massachusetts Chan Medical School, Worcester, the Department of Pathology (J.P., C.K.C., H.L.), the Division of Genetics (J.M.S.), and Department of Cardiology (J.L.), Boston Children's Hospital, and Harvard Medical School (J.P., C.K.C., H.L.), Boston, and Charles River Laboratories, Wilmington (L.B., K. Sutton) - all in Massachusetts; the Department of Clinical Genetics, Institute of Clinical Medicine, University of Tartu (S. Pajusalu), and the Genetics and Personalized Medicine Clinic, Tartu University Hospital (S. Pajusalu) - both in Tartu, Estonia; and Regulatory Innovation, Raleigh, NC (D.B.).
Keith SuttonFrom the Department of Genetics, Yale School of Medicine, New Haven (A.L., K.M., S.H., K.W., S. Pajusalu, M.L.), and Cure Rare Disease, Woodbridge (R.H.) - both in Connecticut; the Departments of Pediatrics (B.W., A.K., R.A., D.K., T.F.) and Neurology (A.R.B.) and Horae Gene Therapy Center and the Li Weibo Institute for Rare Diseases Research (A.K., M.B., K. Sylvia, A.R.B., R.A., D.K., S. Parajuli, T.G., T.F.), University of Massachusetts Chan Medical School, Worcester, the Department of Pathology (J.P., C.K.C., H.L.), the Division of Genetics (J.M.S.), and Department of Cardiology (J.L.), Boston Children's Hospital, and Harvard Medical School (J.P., C.K.C., H.L.), Boston, and Charles River Laboratories, Wilmington (L.B., K. Sutton) - all in Massachusetts; the Department of Clinical Genetics, Institute of Clinical Medicine, University of Tartu (S. Pajusalu), and the Genetics and Personalized Medicine Clinic, Tartu University Hospital (S. Pajusalu) - both in Tartu, Estonia; and Regulatory Innovation, Raleigh, NC (D.B.).
Richard HorganFrom the Department of Genetics, Yale School of Medicine, New Haven (A.L., K.M., S.H., K.W., S. Pajusalu, M.L.), and Cure Rare Disease, Woodbridge (R.H.) - both in Connecticut; the Departments of Pediatrics (B.W., A.K., R.A., D.K., T.F.) and Neurology (A.R.B.) and Horae Gene Therapy Center and the Li Weibo Institute for Rare Diseases Research (A.K., M.B., K. Sylvia, A.R.B., R.A., D.K., S. Parajuli, T.G., T.F.), University of Massachusetts Chan Medical School, Worcester, the Department of Pathology (J.P., C.K.C., H.L.), the Division of Genetics (J.M.S.), and Department of Cardiology (J.L.), Boston Children's Hospital, and Harvard Medical School (J.P., C.K.C., H.L.), Boston, and Charles River Laboratories, Wilmington (L.B., K. Sutton) - all in Massachusetts; the Department of Clinical Genetics, Institute of Clinical Medicine, University of Tartu (S. Pajusalu), and the Genetics and Personalized Medicine Clinic, Tartu University Hospital (S. Pajusalu) - both in Tartu, Estonia; and Regulatory Innovation, Raleigh, NC (D.B.).
Monkol LekFrom the Department of Genetics, Yale School of Medicine, New Haven (A.L., K.M., S.H., K.W., S. Pajusalu, M.L.), and Cure Rare Disease, Woodbridge (R.H.) - both in Connecticut; the Departments of Pediatrics (B.W., A.K., R.A., D.K., T.F.) and Neurology (A.R.B.) and Horae Gene Therapy Center and the Li Weibo Institute for Rare Diseases Research (A.K., M.B., K. Sylvia, A.R.B., R.A., D.K., S. Parajuli, T.G., T.F.), University of Massachusetts Chan Medical School, Worcester, the Department of Pathology (J.P., C.K.C., H.L.), the Division of Genetics (J.M.S.), and Department of Cardiology (J.L.), Boston Children's Hospital, and Harvard Medical School (J.P., C.K.C., H.L.), Boston, and Charles River Laboratories, Wilmington (L.B., K. Sutton) - all in Massachusetts; the Department of Clinical Genetics, Institute of Clinical Medicine, University of Tartu (S. Pajusalu), and the Genetics and Personalized Medicine Clinic, Tartu University Hospital (S. Pajusalu) - both in Tartu, Estonia; and Regulatory Innovation, Raleigh, NC (D.B.).
Terence FlotteFrom the Department of Genetics, Yale School of Medicine, New Haven (A.L., K.M., S.H., K.W., S. Pajusalu, M.L.), and Cure Rare Disease, Woodbridge (R.H.) - both in Connecticut; the Departments of Pediatrics (B.W., A.K., R.A., D.K., T.F.) and Neurology (A.R.B.) and Horae Gene Therapy Center and the Li Weibo Institute for Rare Diseases Research (A.K., M.B., K. Sylvia, A.R.B., R.A., D.K., S. Parajuli, T.G., T.F.), University of Massachusetts Chan Medical School, Worcester, the Department of Pathology (J.P., C.K.C., H.L.), the Division of Genetics (J.M.S.), and Department of Cardiology (J.L.), Boston Children's Hospital, and Harvard Medical School (J.P., C.K.C., H.L.), Boston, and Charles River Laboratories, Wilmington (L.B., K. Sutton) - all in Massachusetts; the Department of Clinical Genetics, Institute of Clinical Medicine, University of Tartu (S. Pajusalu), and the Genetics and Personalized Medicine Clinic, Tartu University Hospital (S. Pajusalu) - both in Tartu, Estonia; and Regulatory Innovation, Raleigh, NC (D.B.).
University of Massachusetts Chan Medical School · USBoston Children's Hospital · USYale University · USCharles River Laboratories (United States) · USTartu University Hospital · EE
Funding
Vector Immunology CoreP01HL158506 · NHLBI · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI Dan Wang · 2021 to 2026
$19.5MNHLBI NIH HHS P01 HL158506
Abstract
We treated a 27-year-old patient with Duchenne's muscular dystrophy (DMD) with recombinant adeno-associated virus (rAAV) serotype 9 containing d
Indexed as
DystrophinGenetic TherapyMuscular Dystrophy, DuchenneRespiratory Distress SyndromeTransgenesAdultAntibodiesFatal OutcomeHumansImmunity, InnateAntibodiesDystrophin
Identifiers
What OpenQuestion holds
Textmetadata
LicenceTDM
Read underepoch 390
Registered trials
None linked
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.