The Bespoke Gene Therapy Consortium: facilitating development of AAV gene therapies for rare diseases

被引：6

作者：

Brooks, P. J. ^{[1
]}

Miller, Timothy M. ^{[2
]}

Revah, Frederic ^{[3
]}

Suh, Junghae ^{[4
]}

Garrison, Bradley R. ^{[5
]}

Starke, Lawrence C. ^{[6
]}

MacLachlan, Timothy K. ^{[7
]}

Neilan, Edward G. ^{[8
]}

Raychaudhuri, Gopa ^{[9
]}

Kassim, Sadik H. ^{[10
]}

Dehdashti, Jean ^{[1
]}

Rutter, Joni L. ^{[1
]}

机构：

[1] NIH, Natl Ctr Adv Translat Sci, Bethesda, MD 20892 USA

[2] Thermo Fisher Sci, Waltham, MA USA

[3] Genethon, Evry, France

[4] Biogen Inc, Cambridge, MA USA

[5] Fdn Natl Inst Hlth, North Bethesda, MD USA

[6] Novartis Pharmaceut, E Hanover, NJ USA

[7] Novartis, Biomed Res, Cambridge, MA USA

[8] Natl Org Rare Disorders, Quincy, MA USA

[9] US FDA, Ctr Biol Evaluat & Res, Silver Spring, MD USA

[10] Danaher Corp, Washington, DC USA

来源：

NATURE REVIEWS DRUG DISCOVERY | 2024年 / 23卷 / 03期

关键词：

D O I：

10.1038/d41573-024-00020-8

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

A public-private partnership managed by the FNIH aims to address biological, manufacturing and regulatory challenges to the development of gene therapies for rare diseases. A public-private partnership managed by the FNIH aims to address biological, manufacturing and regulatory challenges to the development of gene therapies for rare diseases.

引用

页码：157 / 158

页数：2

共 50 条

[21] The emergence of gene therapy for rare diseases
Phillips, M. Ian
Burns, Andrew B.
EXPERT OPINION ON ORPHAN DRUGS, 2014, 2 (11): : 1197 - 1209
[22] Advances in Gene Therapy for Rare Diseases: Targeting Functional Haploinsufficiency Through AAV and mRNA Approaches
Bara-Ledesma, Nuria
Viteri-Noel, Adrian
Lopez Rodriguez, Monica
Stamatakis, Konstantinos
Fabregate, Martin
Vazquez-Santos, Almudena
Gomez del Olmo, Vicente
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES, 2025, 26 (02)
[23] Development of AAV gene therapy for Morquio A syndrome
Tomatsu, S.
FEBS OPEN BIO, 2019, 9 : 31 - 31
[24] Development of Systemic AAV Gene Therapies for Neuronopathic Gaucher Disease
Han, Tae Un
Head, PamelaSara
Gleason, Adenrele
Berhe, Bahafta
Kulkarni, Akhil
Chen, Tiffany
Venditti, Charles P.
Sidransky, Ellen
MOLECULAR THERAPY, 2024, 32 (04) : 523 - 523
[25] Advances and Challenges in the Development of Gene Therapy Medicinal Products for Rare Diseases
Bueren, Juan A.
Auricchio, Alberto
HUMAN GENE THERAPY, 2023, 34 (17-18) : 763 - 775
[26] The clinical landscape for AAV gene therapies
Kuzmin, Dmitry A.
Shutova, Maria V.
Johnston, Natalie R.
Smith, Owen P.
Fedorin, Vasily V.
Kukushkin, Yury S.
van der Loo, Johannes C. M.
Johnstone, Elaine C.
NATURE REVIEWS DRUG DISCOVERY, 2021, 20 (03) : 173 - 174
[27] AAV-Mediated Gene Therapy for the Treatment of Retinal Diseases
Rolling, Fabienne
CURRENT GENE THERAPY, 2010, 10 (05) : 318 - U4
[28] AAV Vectors for Gene Therapy of the Brain in Lysosomal Storage Diseases
Heard, J. M.
HUMAN GENE THERAPY, 2010, 21 (05) : 642 - 642
[29] Management of Neuroinflammatory Responses to AAV-Mediated Gene Therapies for Neurodegenerative Diseases
Perez, Barbara A.
Shutterly, Alison
Chan, Ying Kai
Byrne, Barry J.
Corti, Manuela
BRAIN SCIENCES, 2020, 10 (02)
[30] Immunogenicity assessment of AAV-based gene therapies: An IQ consortium industry white paper
Yang, Tong-yuan
Braun, Manuela
Lembke, Wibke
McBlane, Fraser
Kamerud, John
DeWall, Stephen
Tarcsa, Edit
Fang, Xiaodong
Hofer, Lena
Kavita, Uma
Upreti, Vijay V.
Gupta, Swati
Loo, LiNa
Johnson, Alison J.
Chandode, Rakesh Kantilal
Stubenrauch, Kay-Gunnar
Vinzing, Maya
Xia, Cindy Q.
Jawa, Vibha
MOLECULAR THERAPY METHODS & CLINICAL DEVELOPMENT, 2022, 26 : 471 - 494

← 1 2 3 4 5 →