Human Gene Therapy, volume 36, issue 5-6, pages 653-662
Adeno-Associated Virus Gene Therapy Development: Early Planning and Regulatory Considerations to Advance the Platform Vector Gene Therapy Program
Richa Madan Lomash
1
,
Jean DEHDASHTI
2
,
Oleg A. Shchelochkov
3
,
Randy J. Chandler
3
,
Lina Li
3
,
Irini Manoli
3
,
Jennifer L. Sloan
3
,
Pramod Terse
1
,
Xin Xu
1
,
Dimah Saade
4
,
Rodica Stan
1
,
Philip J. Brooks
2
,
Donald C. Lo
1
,
Carsten G. Bönnemann
4
,
Charles P. Venditti
3
,
Anne R. Pariser
2
,
Elizabeth A. Ottinger
1
2
Division of Rare Diseases Research and Innovation, NCATS, NIH, Rockville, Maryland, USA
3
Organic Acid Research Section, Metabolic Medicine Branch, National Human Genome Research Institute (NHGRI), NIH, Bethesda, Maryland, USA
4
Neuromuscular and Neurogenetic Disorders of Childhood Section, National Institute of Neurological Disorders and Stroke (NINDS), NIH, Bethesda, Maryland, USA.
Publication type: Journal Article
Publication date: 2025-03-01
Journal:
Human Gene Therapy
scimago Q2
SJR: 1.091
CiteScore: 6.5
Impact factor: 3.9
ISSN: 10430342, 15577422
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