Discover Community Create You

Loading publication...

Noodle always counts identifier-free feature events for service operation. With your permission, Google Analytics can also estimate audience and engagement. Search text, publication identifiers, cookies, IP addresses and browser fingerprints are never included in Noodle's first-party event counters.

Privacy details

Article

Limb‐girdle muscular dystrophy type 2D gene therapy restores α‐sarcoglycan and associated proteins

16 Apr 2009

Abstract excerpt

OBJECTIVE: alpha-Sarcoglycan deficiency results in a severe form of muscular dystrophy (limb-girdle muscular dystrophy type 2D [LGMD2D]) without treatment. Gene replacement represents a strategy for correcting the underlying defect. Questions related to this approach were addressed in this clinical trial, particularly the need for immunotherapy and persistence of gene expression. METHODS: A double-blind,...

Read the complete abstract on PubMed

Topics

Share this publication in a Topic to start or enrich a Post.

Share to Muscle Physiology and Disorders
Share to Virus-based gene therapy research
Share to Xenotransplantation and immune response

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Source

Identifiers

PMID: 19798725
DOI: 10.1002/ana.21732

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 3 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Limb‐girdle muscular dystrophy type 2D gene therapy restores α‐sarcoglycan and associated proteinsPMID 19798725Read ↗

Sustained alpha‐sarcoglycan gene expression after gene transfer in limb‐girdle muscular dystrophy, type 2DSemantic proximity Read ↗

A phase I trial of adeno-associated virus serotype 1-γ-sarcoglycan gene therapy for limb girdle muscular dystrophy type 2CSemantic proximity Read ↗

β-Sarcoglycan gene transfer decreases fibrosis and restores force in LGMD2E mice.Semantic proximity Read ↗

Phenotypic correction of alpha-sarcoglycan deficiency by intra-arterial injection of a muscle-specific serotype 1 rAAV vector.Semantic proximity Read ↗

Gene therapy with bidridistrogene xeboparvovec for limb-girdle muscular dystrophy type 2E/R4: phase 1/2 trial resultsSemantic proximity Read ↗

Long-term Skeletal Muscle Protection After Gene Transfer in a Mouse Model of LGMD-2DSemantic proximity Read ↗

Progress and prospects of gene therapy clinical trials for the muscular dystrophiesSemantic proximity Read ↗

Systemic γ-sarcoglycan AAV gene transfer results in dose-dependent correction of muscle deficits in the LGMD 2C/R5 mouse modelSemantic proximity Read ↗

AAV microdystrophin gene replacement therapy for Duchenne muscular dystrophy: progress and prospectsSemantic proximity Read ↗

Muscle-Specific Promoters May Be Necessary for Adeno-Associated Virus-Mediated Gene Transfer in the Treatment of Muscular DystrophiesSemantic proximity Read ↗

Rescue of skeletal muscles of gamma-sarcoglycan-deficient mice with adeno-associated virus-mediated gene transfer.Semantic proximity Read ↗

Adeno-Associated Virus (AAV)-Mediated Gene Therapy for Duchenne Muscular Dystrophy: The Issue of Transgene PersistenceSemantic proximity Read ↗

⌁Select a neighboring publication to make it the new centre.