AI Article Synopsis
- The review aims to create a framework for understanding various molecular treatments for muscular dystrophy.
- Recent advancements include the approval of antisense oligonucleotides for Duchenne muscular dystrophy and spinal muscular atrophy, along with ongoing clinical trials using recombinant adeno-associated virus.
- This progress indicates that we may soon be able to effectively treat and potentially cure muscular dystrophies through a better understanding of their molecular basis and treatment strategies.
Article Abstract
Purpose Of Review: To construct a framework to understand the different molecular interventions for muscular dystrophy.
Recent Findings: The recent approval of antisense oligonucleotides treatment for Duchenne muscular dystrophy and spinal muscular atrophy and current clinical trials using recombinant adeno-associated virus for the treatment of those diseases suggests that we are at a tipping point where we are able to treat and potentially cure muscular dystrophies. Understanding the basic molecular pathogenesis of muscular dystrophies and the molecular biology of the treatment allows for critical evaluation of the proposed therapies.
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| http://dx.doi.org/10.1007/s11940-018-0509-2 | DOI Listing |
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