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- Principles and general themes
-
1. Oncolytic viruses: strategies, applications and challenges
- Dr. Stephen J. Russell
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2. Directed evolution of AAV delivery systems for clinical gene therapy
- Prof. David Schaffer
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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
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13. Surface-mediated targeting of lentiviral vectors
- Prof. Dr. Christian Buchholz
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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
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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
- Overview (1)
- Stem cell gene therapy
- Examples of gene correction in stem cells
- Methods for monitoring gene transfer
- Technology for mapping integration sites
- Next-gen sequencing technologies
- Lab databases
- HIV integration targeting
- Sites of HIV-1 cDNA integration in SupT1 cells
- Integrase is a viral determinant of targeting
- PSIP1/LEDGF/p75 in HIV integration targeting
- Depletion of LEDGE/p75 redirects integration
- LEDGF/p75 expression and integration frequency
- Fusions of HP1β(CBX) to LEDGF/p75
- Integration in transcription units
- Integration in presence of HP1-LEDGF fusion
- Some LEDGF/p75 fusions studied to date
- HIV integration targeting: summary
- Gammaretroviral integration targeting
- Integrase: viral determinant of integration targeting
- Retrovirus integration & histone methylation (1)
- Retrovirus integration & histone methylation (2)
- Retrovirus integration & histone acetylation
- Retrovirus integration relative to bound proteins
- BET proteins: tethering factors for MLV integrase
- Modulating BET protein binding with JQ1
- MLV integration and BET proteins
- Gammaretroviral integration targeting: summary
- Vector integration in human gene therapy
- Mechanisms of insertional activation
- Lentiviral vectors for gene therapy
- Beta-thalassemia: lentiviral gene therapy (1)
- Beta-thalassemia: lentiviral gene therapy (2)
- Integration site analysis
- Mechanisms of insertional activation
- HMGA2
- Genotoxicity in SCID-X1 (1)
- Genotoxicity in SCID-X1 (2)
- Clonal expansion not predictor of adverse events
- Vector integration in gene therapy: summary
- Challenges in using integration site data
- Breaking DNA more randomly
- Breaking DNA more randomly: Jargon
- Multiple adaptors to suppress contamination
- Marking integration sites pre-PCR for analysis
- The purpose of integration site monitoring
- Analysis of integration sites in adverse events
- Overview (2)
- Credits
Topics Covered
- Introduction: human gene therapy with integrating vectors
- Methods for monitoring gene transfer
- HIV integration targeting
- Gammaretroviral integration targeting
- Analysis of vector integration in human gene therapy
Links
Series:
Categories:
Therapeutic Areas:
Talk Citation
Bushman, F. (2014, August 5). Gene therapy and gene transfer: vector integration preferences and integration site analysis [Video file]. In The Biomedical & Life Sciences Collection, Henry Stewart Talks. Retrieved November 9, 2024, from https://doi.org/10.69645/CEZJ6131.Export Citation (RIS)
Publication History
Financial Disclosures
- Prof. Frederic Bushman has not informed HSTalks of any commercial/financial relationship that it is appropriate to disclose.
Gene therapy and gene transfer: vector integration preferences and integration site analysis
Published on August 5, 2014
50 min
A selection of talks on Genetics & Epigenetics
Transcript
Please wait while the transcript is being prepared...
0:00
My name is Frederic Bushman.
I'm a professor at the
University of Pennsylvania.
And the title of this lecture is
Vector Integration Preferences
and Integration Site Analysis.
0:12
This lecture will begin with an
introduction, just a little bit
about gene therapy and
vector integration.
Then methods for
monitoring gene transfer.
Then HIV integration targeting.
Then gammaretroviral integration
targeting, which differs.
And lastly, analysis of vector
integration in human gene therapy.
0:35
Next slide shows the general
idea of stem cell gene therapy
to treat an inherited disease.
So bone marrow cells are
removed from the patient,
and then gene-corrected
by transduction ex vivo
with retroviral vector that
encodes the corrective gene, shown
in the upper left.
So that installs the corrective
gene into bone marrow cells,
lower left, where each cell
now has the vector integrated
at a different location
in the human genome.
You can then re-infuse those
cells into the subject.
And bone marrow cells
produce daughter
cells, which circulate in the blood.
You can then sample
cells from blood years
later, shown on the
right, and sequence
integration site distributions.
And that can tell you things like
how many different kinds of cells;
how many different progenitor
cells or contributing cells
to the periphery; and whether
some cells are contributing more
than others; and in cases
of adverse events, leukemia,
you can tell where the
integration site was
and get some information
on the genes involved.
So stem cell gene therapy has been
used to treat many subjects now
with inherited immuno-deficiencies
and other conditions.
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