Moving along....

https://musculardystrophynews.com/news/galgt2-gene-therapy-aids-dmd-boys-muscle-function-stability/



"The therapy was deemed safe in a preclinical study at Nationwide Children’s Hospital. Further studies have shown that increasing GALGT2 levels prevented muscle injury in a mouse model of DMD. It also prevented decline in heart function as DMD mice aged. Studies in mice have supported its potential in limb girdle muscular dystrophy and congenital muscular dystrophy."

Sarepta Therapeutics‘ investigational GALGT2 gene therapy helped stabilize muscle function when delivered at a higher dose to a boy with Duchenne muscular dystrophy (DMD), data from a Phase 1/2a clinical trial show.


Unlike other experimental approaches that deliver a portion of the DMD gene, this surrogate gene therapy (known as rAAVrh74.MCK.GALGT2) carries a copy of the GALGT2 gene — also known as B4GALNT2 — that’s been shown to induce production or alterations to proteins that inhibit muscular dystrophy from developing.


Repeat that: "...that’s been shown to induce production or alterations to proteins that inhibit muscular dystrophy from developing."


The study, “A first-in-human phase I/IIa gene transfer clinical trial for Duchenne muscular dystrophy using rAAVrh74.MCK.GALGT2,” was published in Molecular Therapy: Methods & Clinical Development.

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9483573/

"....First, overexpression of GALGT2 not only induces glycosylation of α-dystroglycan but increases the ectopic expression of its normally synaptic binding partners in both mdx and wild-type skeletal muscles, and overexpression of many of these partners has been shown to inhibit the development of muscular dystrophy.12,25, 26, 27 Second, GALGT2 overexpression prevents eccentric contraction-induced muscle injury in both dystrophic mdx and wild-type mouse muscles.11,26 Third, GALGT2 overexpression in mdx mouse heart can prevent the loss of cardiac function as the mice age, a finding of potentially significant clinical relevance in the DMD population.18 Finally, GALGT2 overexpression has been shown to inhibit the development of muscle pathology in mouse models of four different forms of muscular dystrophy, the mdx model of DMD,11,12,14,17 the dyW model of congenital muscular dystrophy 1A (MDC1A),15 the Sgca−/− model of limb-girdle muscular dystrophy 2D (LGMD2D),16 and the FKRPP448L model of limb-girdle muscular dystrophy 2I (LGMD2I),13 raising the possibility of a membrane-stabilizing therapeutic that is broadly applicable to different genetic MD forms."