Evidence map›Paper›PMID 42137291›Full record

ArticleMolecular therapy. Advances2026

Preclinical evaluation of INS1201 AAV9-micro-dystrophin via CSF administration as a potential therapy for Duchenne muscular dystrophy.

Gretchen Thomsen, Allan Kaspar, Laura Ferraiuolo, Binh Chu, Veronica J Garcia, Ryan Weiss, Theresa Slaiwa, Nuria Casanova-Vallve, Robert Cano, Erin Hurley and 26 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy. Advances, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

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.

2 · The registry

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.

3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

36 authors.

Gretchen ThomsenInsmed Gene Therapy LLC, San Diego, CA 92121, USA.
Allan KasparInsmed Gene Therapy LLC, San Diego, CA 92121, USA.
Laura FerraiuoloInsmed Gene Therapy LLC, San Diego, CA 92121, USA.
Binh ChuInsmed Gene Therapy LLC, San Diego, CA 92121, USA.
Veronica J GarciaInsmed Gene Therapy LLC, San Diego, CA 92121, USA.
Ryan WeissInsmed Gene Therapy LLC, San Diego, CA 92121, USA.
Theresa SlaiwaInsmed Gene Therapy LLC, San Diego, CA 92121, USA.
Nuria Casanova-VallveInsmed Gene Therapy LLC, San Diego, CA 92121, USA.
Robert CanoInsmed Gene Therapy LLC, San Diego, CA 92121, USA.
Erin HurleyInsmed Gene Therapy LLC, San Diego, CA 92121, USA.
Hana JulazadehInsmed Gene Therapy LLC, San Diego, CA 92121, USA.
Haunani BautistaInsmed Gene Therapy LLC, San Diego, CA 92121, USA.
Ashley RenteriaInsmed Gene Therapy LLC, San Diego, CA 92121, USA.
Maiya LeeInsmed Gene Therapy LLC, San Diego, CA 92121, USA.
Jaedyn RollinsInsmed Gene Therapy LLC, San Diego, CA 92121, USA.
Melissa McAlonis-DownesDepartment of Cellular and Molecular Medicine, University of California, San Diego, La Jolla, CA 92093, USA.
W David ArnoldUniversity of Missouri, Columbia, MO 65211, USA.
Tracy TreDenickBioTechLogic, LLC, a Dark Horse Consulting Group, Inc. Company, Walnut Creek, CA 94597, USA.
Ryan HsuSalk Institute for Biological Studies, La Jolla, CA 92037, USA.
Lynn E BaylessInsmed Gene Therapy LLC, San Diego, CA 92121, USA.
Catherine KozialInsmed Gene Therapy LLC, San Diego, CA 92121, USA.
Christopher ForespringInsmed Gene Therapy LLC, San Diego, CA 92121, USA.
Michael MolonyInsmed Gene Therapy LLC, San Diego, CA 92121, USA.
Michael FraserInsmed Gene Therapy LLC, San Diego, CA 92121, USA.
Junguo ZhouInsmed Incorporated, Bridgewater, NJ 08807, USA.
Jiaqin YaoInsmed Incorporated, Bridgewater, NJ 08807, USA.
Shannon N BremnerDepartment of Orthopaedic Surgery, School of Medicine, University of California, San Diego, La Jolla, CA 92037, USA.
Samuel R WardDepartment of Orthopaedic Surgery, School of Medicine, University of California, San Diego, La Jolla, CA 92037, USA.
Simon SchenkDepartment of Orthopaedic Surgery, School of Medicine, University of California, San Diego, La Jolla, CA 92037, USA.
Tetsuya YoshikawaDrug Safety Research Laboratories, Shin Nippon Biomedical Laboratories, Ltd., Kagoshima 891-1394, Japan.
Akiko MoriyamaDrug Safety Research Laboratories, Shin Nippon Biomedical Laboratories, Ltd., Kagoshima 891-1394, Japan.
Hideshi TsusakiDrug Safety Research Laboratories, Shin Nippon Biomedical Laboratories, Ltd., Kagoshima 891-1394, Japan.
Samit VarmaInsmed Gene Therapy LLC, San Diego, CA 92121, USA.
Marissa KrugerInsmed Gene Therapy LLC, San Diego, CA 92121, USA.
Mark StahlInsmed Gene Therapy LLC, San Diego, CA 92121, USA.
Brian KasparInsmed Gene Therapy LLC, San Diego, CA 92121, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

INS1201 is an investigational adeno-associated virus 9-micro-dystrophin gene transfer therapy under development as a potential treatment for Duchenne muscular dystrophy (DMD). Administered into the cerebrospinal fluid (CSF), this delivery approach efficiently targets skeletal and cardiac muscle. Here, we present preclinical findings that demonstrated improved muscle histopathology and functional efficacy in the Video Abstract:

Indexed as

AAV9Duchenne muscular dystrophygene therapyintrathecalmdxmicro-dystrophinmouse modelnonhuman primatespreclinicalrecombinant adeno-associated virus

Identifiers

PMID42137291
PMCPMC13148950

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
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.