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Printable Handouts
Navigable Slide Index
- Introduction
- Duchenne muscular dystrophy (DMD) (1)
- Duchenne muscular dystrophy (DMD) (2)
- Characteristics of dystrophic muscle
- Becker muscular dystrophy (BMD)
- Affected femails
- The dystrophin gene
- Dystrophin structure
- Dystrophin isoforms
- Dystrophin (1)
- Dystrophin (2)
- The dystrophin-glycoprotein complex
- Dystrophin associated proteins in MDs
- Molecular basis of DMD/BMD
- DMD vs. BMD
- Animal models for DMD
- Potential approaches to a therapy for DMD
- Potential for gene therapy of DMD
- Strategies for development of DMD gene therapy
- Viral gene transfer to muscle
- Adeno-associated viruses (AVV)
- Development of functional micro-dystrophins
- Predicted structure/function of truncated dystrophin
- Assembly of dystrophin-complex by micro-dys
- Microdystrophin-AVV vectors
- Co-transfection of transfer and helper plasmids
- How can a gene be transferred to all muscle cells?
- Systemic gene transfer to mouse muscles
- Dystrophin injected mouse muscles
- Long-term gene expression after AVV delivery (1)
- Long-term gene expression after AVV delivery (2)
- Dystrophin/utrophin -/- mice (mdx:utrn -/-)
- Appearance of mdx vs. mdx:utrn -/- (dko) mice
- Quadriceps histopathology in mdx vs. dko mice
- Immunostaing 1 year after dystrophin injection
- Treated dko mice exhibit increased muscle mass
- Muscle function - diaphragm
- Mortality and body mass: mdx:utrn -/-
- Transplicing AVV vectors for larger proteins
- Transcript modification using anti-sense oligos
- Exon skipping via direct delivery
- Multiple approaches for gene therapy of MDs
- References
Topics Covered
- Introduction to duchenne muscular dystrophy (DMD)
- Dystrophin, the gene product of the DMD locus
- Structure of dystrophin
- The dystrophin glycoprotein complex
- Gene therapy for DMD
- Adeno-associated viral (AAV) vectors
- Mini- and micro-dystrophins
- Delivery of AAV vectors to muscles body-wide
- Gene therapy of muscular dystrophy in mouse models using AAV
- Alternative approaches to gene therapy of DMD
Links
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Talk Citation
Chamberlain, J. (2007, October 1). Duchenne muscular dystrophy [Video file]. In The Biomedical & Life Sciences Collection, Henry Stewart Talks. Retrieved December 5, 2024, from https://doi.org/10.69645/ORRY4669.Export Citation (RIS)
Publication History
Financial Disclosures
- Prof. Jeff Chamberlain has not informed HSTalks of any commercial/financial relationship that it is appropriate to disclose.