Evidence map›Paper›PMID 40868246›Full record

ReviewBiomedicines2025

Engineering Targeted Gene Delivery Systems for Primary Hereditary Skeletal Myopathies: Current Strategies and Future Perspectives.

Jiahao Wu, Yimin Hua, Yanjiang Zheng, Xu Liu, Yifei Li

Abstract readReview
PubMed Full text at PMC Publisher
In one paragraph

Review in Biomedicines, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing 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

2 citing papers in PubMed.

  1. Review
  2. Review
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

5 authors.

Jiahao WuKey Laboratory of Birth Defects and Related Diseases of Women and Children of MOE, NHC Key Laboratory of Chronobiology, Department of Pediatrics, West China Second University Hospital, Sichuan University, Chengdu 610041, China.
Yimin HuaKey Laboratory of Birth Defects and Related Diseases of Women and Children of MOE, NHC Key Laboratory of Chronobiology, Department of Pediatrics, West China Second University Hospital, Sichuan University, Chengdu 610041, China.
Yanjiang ZhengKey Laboratory of Birth Defects and Related Diseases of Women and Children of MOE, NHC Key Laboratory of Chronobiology, Department of Pediatrics, West China Second University Hospital, Sichuan University, Chengdu 610041, China.
Xu LiuKey Laboratory of Birth Defects and Related Diseases of Women and Children of MOE, NHC Key Laboratory of Chronobiology, Department of Pediatrics, West China Second University Hospital, Sichuan University, Chengdu 610041, China.
Yifei LiKey Laboratory of Birth Defects and Related Diseases of Women and Children of MOE, NHC Key Laboratory of Chronobiology, Department of Pediatrics, West China Second University Hospital, Sichuan University, Chengdu 610041, China.ORCID 0000-0002-3096-4287

Funding

National Natural Science Foundation of China 82270249National Natural Science Foundation of China 82470249
6 · The paper itself

Abstract

Skeletal muscle, constituting ~40% of body mass, serves as a primary effector for movement and a key metabolic regulator through myokine secretion. Hereditary myopathies, including dystrophinopathies (DMD/BMD), limb-girdle muscular dystrophies (LGMD), and metabolic disorders like Pompe disease, arise from pathogenic mutations in structural, metabolic, or ion channel genes, leading to progressive weakness and multi-organ dysfunction. Gene therapy has emerged as a transformative strategy, leveraging viral and non-viral vectors to deliver therapeutic nucleic acids. Adeno-associated virus (AAV) vectors dominate clinical applications due to their efficient transduction of post-mitotic myofibers and sustained transgene expression. Innovations in AAV engineering, such as capsid modification (chemical conjugation, rational design, directed evolution), self-complementary genomes, and tissue-specific promoters (e.g., MHCK7), enhance muscle tropism while mitigating immunogenicity and off-target effects. Non-viral vectors (liposomes, polymers, exosomes) offer advantages in cargo capacity (delivering full-length dystrophin), biocompatibility, and scalable production but face challenges in transduction efficiency and endosomal escape. Clinically, AAV-based therapies (e.g., Elevidys

Indexed as

AAV vector engineeringgene therapyskeletal muscle diseaseskeletal muscle target

Identifiers

PMID40868246
PMCPMC12383629

What Socratic holds

Textmetadata
LicenceCC BY
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.