New hope for older SMA patients with next-generation self-complementary AAV gene therapy

Hiroyuki Nakai; Erika L. Finanger; Richard Jude Samulski

doi:10.1016/j.ymthe.2025.09.013

New hope for older SMA patients with next-generation self-complementary AAV gene therapy

Hiroyuki Nakai
, Erika L. Finanger
, Richard Jude Samulski

Research output: Contribution to journal › Comment/debate › peer-review

Original language	English (US)
Pages (from-to)	4662-4664
Number of pages	3
Journal	Molecular Therapy
Volume	33
Issue number	10
DOIs	https://doi.org/10.1016/j.ymthe.2025.09.013
State	Published - Oct 1 2025

ASJC Scopus subject areas

Molecular Medicine
Molecular Biology
Genetics
Pharmacology
Drug Discovery

Access to Document

10.1016/j.ymthe.2025.09.013

Cite this

@article{6352ddb37a024322a600d60c05dce333,

title = "New hope for older SMA patients with next-generation self-complementary AAV gene therapy",

author = "Hiroyuki Nakai and Finanger, \{Erika L.\} and Samulski, \{Richard Jude\}",

year = "2025",

month = oct,

day = "1",

doi = "10.1016/j.ymthe.2025.09.013",

language = "English (US)",

volume = "33",

pages = "4662--4664",

journal = "Molecular Therapy",

issn = "1525-0016",

publisher = "Cell Press",

number = "10",

}

TY - JOUR

T1 - New hope for older SMA patients with next-generation self-complementary AAV gene therapy

AU - Nakai, Hiroyuki

AU - Finanger, Erika L.

AU - Samulski, Richard Jude

PY - 2025/10/1

Y1 - 2025/10/1

UR - https://www.scopus.com/pages/publications/105016811828

UR - https://www.scopus.com/pages/publications/105016811828#tab=citedBy

U2 - 10.1016/j.ymthe.2025.09.013

DO - 10.1016/j.ymthe.2025.09.013

M3 - Comment/debate

C2 - 40987285

AN - SCOPUS:105016811828

SN - 1525-0016

VL - 33

SP - 4662

EP - 4664

JO - Molecular Therapy

JF - Molecular Therapy

IS - 10

ER -

New hope for older SMA patients with next-generation self-complementary AAV gene therapy

ASJC Scopus subject areas

Access to Document

Other files and links

Cite this