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- Principles and general themes
-
1. Oncolytic viruses: strategies, applications and challenges
- Dr. Stephen J. Russell
-
2. Directed evolution of AAV delivery systems for clinical gene therapy
- Prof. David Schaffer
-
6. The host response: adaptive immune response to viral vector delivery
- Prof. Roland W. Herzog
-
7. Gene therapy and virotherapy in the treatment of cancer
- Prof. Leonard Seymour
-
8. Gene therapy for the muscular dystrophies
- Prof. Jeff Chamberlain
- Major gene transfer platforms and gene therapy strategies
-
9. Gammaretroviral vectors: biology, design and applications
- Prof. Axel Schambach
-
13. Surface-mediated targeting of lentiviral vectors
- Prof. Dr. Christian Buchholz
-
14. Gene transfer and gene therapy
- Dr. David A. Williams
-
15. Tracking vector insertion sites to explore the biology of transduced cells in vivo
- Prof. Dr. Christof Von Kalle
-
16. Advances in gene therapy for respiratory diseases 1
- Prof. John F. Engelhardt
-
17. Advances in gene therapy for respiratory diseases 2
- Prof. John F. Engelhardt
-
20. Gene therapy for hemophilia
- Prof. Katherine High
- New technologies for sequence-specific editing of gene expression
-
21. Helper-dependent adenoviral vectors for gene therapy
- Prof. Nicola Brunetti-Pierri
-
22. HSV vectors: approaches to the treatment of chronic pain
- Prof. Joseph C. Glorioso
- Archived Lectures *These may not cover the latest advances in the field
-
23. RNAi for neurological diseases
- Prof. Beverly L. Davidson
-
24. Directed evolution of novel adeno-associated viral vectors for gene therapy
- Prof. David Schaffer
Printable Handouts
Navigable Slide Index
- Introduction
- The muscular dystrophies
- More than 50 known types of muscular dystrophy
- Proteins defective in muscular dystrophies
- Duchenne muscular dystrophy (DMD) (1)
- Duchenne muscular dystrophy (DMD) (2)
- Presentation of DMD in young boys
- Progressive muscle degeneration in DMD
- Becker muscular dystrophy (BMD)
- The dystrophin gene
- Dystrophin structure
- Dystrophin localization in skeletal muscle
- Molecular basis of DMD/BMD
- DMD vs. BMD
- Gene with exon mutation
- Dystrophin links the actin cytoskeleton to ECM
- Current management of DMD pathology
- Strategies for the development of DMD therapies
- Gene therapy for DMD
- Development of functional micro-dystrophins
- Assembly of the dystrophin-complex by micro-Dys
- AAV vectors for dystrophin delivery
- Vectors based on AAV serotype 6 & gene delivery
- Expression of micro-Dys one year after injection
- Scaling systemic delivery to larger subjects
- Sustained expression of canine micro-dystrophin
- Summary so far
- Transcript modification: anti-sense oligos (ASO)
- Dystrophin expression after AAV-ASO delivery
- Exon skipping via direct delivery of ASO (1)
- Ring linkage and modifications
- Exon skipping via direct delivery of ASO (2)
- Human clinical trials of antisense technology
- Multiple approaches for gene therapy of MDs
- Acknowledgements
Topics Covered
- Introduction to the muscular dystrophies
- Duchenne muscular dystrophy (DMD)
- Dystrophin, the gene product of the DMD locus
- The molecular basis of DMD
- Strategies for the development of DMD therapies
- Adeno-associated viral (AAV) vectors and micro-dystrophins
- Scaling gene therapies to large animal models
- Exon skipping therapies
Links
Series:
Categories:
Therapeutic Areas:
Talk Citation
Chamberlain, J. (2014, September 3). Gene therapy for the muscular dystrophies [Video file]. In The Biomedical & Life Sciences Collection, Henry Stewart Talks. Retrieved December 3, 2024, from https://doi.org/10.69645/HQUB5771.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.
A selection of talks on Neurology
Transcript
Please wait while the transcript is being prepared...
0:00
My
name is Jeff Chamberlain
and I'm at the
University of Washington.
I'm going to discuss gene therapy
for the muscular dystrophies.
Most of my lecture will focus
on Duchenne muscular dystrophy,
which is the most common form.
0:15
The muscular dystrophies are a group
of single-gene inherited disorders.
There are many different types
of muscular dystrophy, several
of which have still
not been identified.
Most of the different types of
muscular dystrophy cannot be
distinguished at a clinical level
and that requires genetic testing
to tell which type
an individual has.
The muscular dystrophies
share the common feature
of having progressive
muscle weakness.
It generally affects the
skeletal or the limb muscles,
but it can also affect
cardiac and smooth muscles.
Sometimes the central nervous
system is involved, particularly
in the early onset or the
congenital muscular dystrophies.
Different types of
muscular dystrophy
can arise either soon
after birth, in childhood,
or in older individuals.
And even though the muscles
are the same in every person,
the different types
of muscular dystrophy
can have different effects
on different muscles.
1:11
As shown in this slide, there
are many different types
of muscular dystrophy characterized
by a variety of different names.
At the top, I've highlighted
Duchenne Muscular Dystrophy,
or DMD, as well as limb-girdle
muscular dystrophy.
These tend to be the most
common of the recessively
inherited the muscular dystrophies.
There are many different types of
limb-girdle muscular dystrophy,
and the list keeps
growing as more genes are
identified as causing
these disorders.
At the bottom of the list
are indicated FSHD and DM.
These represent facioscapulohumeral
muscular dystrophy
and myotonic dystrophy.
These are the two
most common dominantly
inherited muscular dystrophies.
And even though this slide,
which is somewhat old,
indicates that the FSHD
gene was not known.
It has in the last
few years been shown
to be caused by altered copy
number of the DUX4 gene.