Evidence map›Paper›PMID 42003884›Full record

ArticleMolecular therapy. Nucleic acids2026

AAV-mediated gene transfer of a novel microdystrophin ameliorates pathology and enhances muscle function in a mouse model of DMD.

Lawrence Owusu, SunJung Kim, Hiren Patel, Steven Foltz, Gary N Y Chan, Kwi Hye Kim, Aaron Kopen, Lin Yang, Michael W Lawlor, Nicholas Buss and 4 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy. Nucleic acids, 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

14 authors.

Lawrence OwusuREGENXBIO Inc., 9804 Medical Center Drive, Rockville, MD, USA.
SunJung KimREGENXBIO Inc., 9804 Medical Center Drive, Rockville, MD, USA.
Hiren PatelREGENXBIO Inc., 9804 Medical Center Drive, Rockville, MD, USA.
Steven FoltzREGENXBIO Inc., 9804 Medical Center Drive, Rockville, MD, USA.
Gary N Y ChanREGENXBIO Inc., 9804 Medical Center Drive, Rockville, MD, USA.
Kwi Hye KimREGENXBIO Inc., 9804 Medical Center Drive, Rockville, MD, USA.
Aaron KopenREGENXBIO Inc., 9804 Medical Center Drive, Rockville, MD, USA.
Lin YangREGENXBIO Inc., 9804 Medical Center Drive, Rockville, MD, USA.
Michael W LawlorDiverge Translational Science Laboratory, 247 W Freshwater Way, Suite 600, Milwaukee, WI, USA.
Nicholas BussREGENXBIO Inc., 9804 Medical Center Drive, Rockville, MD, USA.
Justine J CunninghamREGENXBIO Inc., 9804 Medical Center Drive, Rockville, MD, USA.
Ye LiuREGENXBIO Inc., 9804 Medical Center Drive, Rockville, MD, USA.
Olivier DanosREGENXBIO Inc., 9804 Medical Center Drive, Rockville, MD, USA.
Michele FiscellaREGENXBIO Inc., 9804 Medical Center Drive, Rockville, MD, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Use of adeno-associated virus (AAV)-mediated transfer of functional microdystrophins to address Duchenne muscular dystrophy (DMD) has been established. RGX-202, an AAV8 vector encoding a novel, optimized human microdystrophin with an extended C-terminal domain, expressed under the Spc5-12 muscle-specific promoter, was evaluated for tolerability and efficacy in dystrophin-deficient

Indexed as

AAV8Duchenne muscular dystrophyefficacyextended C-terminal domaingene therapymicrodystrophinminimum effective doseMT: Clinical ApplicationspharmacologyRGX-202

Identifiers

PMID42003884
PMCPMC13091440

What Socratic 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 Socratic graph.