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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
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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
-
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
- Clinical trials with Ad vectors
- Overview of gene therapy vectors
- Retroviral vectors
- Lentiviral vectors
- Adeno-associated viral vectors
- Non-viral vectors
- Adenoviral vectors
- Human adenovirus
- First generation Ad vector
- Helper-Dependent Ad vector
- Cre/loxP system for generating HDAd
- Large scale production of HDAd
- Pros and cons of HDAd
- HDAd vector applications
- Liver-directed gene therapy
- Inborn errors of metabolism
- First gene therapy study
- Strategies for liver-directed gene therapy
- Ex vivo gene therapy trial with retroviral vectors
- In vivo strategies for liver-directed gene therapy
- HDAd-ApoE in ApoE deficient mice
- HDAd-ApoE in ApoE deficient mice: toxicity
- HDAd-ApoE in ApoE deficient mice: efficacy
- Hemophilia B gene therapy
- HDAd-mediated hemophilia B therapy: efficacy
- HDAd-mediated hemophilia B therapy: toxicity
- Fatal response in treated patient
- OTC deficiency clinical trial: subjects
- OTC deficiency clinical trial: clinical outcome
- OTC deficiency clinical trial: immune response
- OTC deficiency clinical trial: systemic treatment
- HDAd lethality in baboons
- X-gal histochemistry in the baboon study
- Laboratory findings of baboon study
- Ad vector toxicity
- Threshold effect
- Reasons for Ad vector toxicity
- In vivo hepatocyte gene therapy
- Pathways for hepatocyte transduction
- Problems and solutions
- Surgical delivery of HDAd
- Surgical delivery of HDAd: results
- Balloon occlusion catheter-based delivery of HDAd
- Level and duration of transgene expression
- Balloon catheter method: laboratory findings
- Balloon method has higher expression levels
- Balloon catheter delivery of HDAd-hFIX in monkeys
- Summary and conclusions
- Another solution for problems
- HDAd evading Kupffer cells
- HDAd evading Kupffer cells: HDAd5/6
- SR-A and SREC-I co-localize with HDAd
- Prospects for HDAd liver-directed gene therapy
- Lung directed gene therapy
- CF gene therapy
- Transduction efficiency
- HDAd is significantly less toxic than FGAd
- Therapeutic potential of HDAd for CF
- Intracorporeal nebulizing catheter: AeroProbe
- Issues with HDAd-mediated CF gene therapy
- Brain gene therapy
- HDAd as genetic vaccine
- HDAd and stem cells
- Acknowledgements
Topics Covered
- Overview of gene therapy vectors
- Helper-Dependent Ad (HDAd) vector
- Liver and lung directed gene therapy
- Hemophilia B gene therapy
- OTC deficiency clinical trial
- Balloon occlusion catheter-based delivery of HDAd
- HDAd-mediated CF gene therapy
- Brain gene therapy
- HDAd as genetic vaccine
- HDAd and stem cells
Talk Citation
Brunetti-Pierri, N. (2014, August 5). Helper-dependent adenoviral vectors for gene therapy [Video file]. In The Biomedical & Life Sciences Collection, Henry Stewart Talks. Retrieved October 12, 2024, from https://doi.org/10.69645/ZHCF8778.Export Citation (RIS)
Publication History
Financial Disclosures
- Prof. Nicola Brunetti-Pierri has not informed HSTalks of any commercial/financial relationship that it is appropriate to disclose.
A selection of talks on Genetics & Epigenetics
Transcript
Please wait while the transcript is being prepared...
0:00
I want to welcome you
to this presentation.
My name is Nicola Brunetti-Pierri.
And I work as an
Associate Investigator
at the Telethon Institute
of Genetics and Medicine.
I'm also affiliated with the
Department of Translational
Medicine of Federico II
University in Naples, Italy.
In this talk, I would like
to bring your attention
to helper-dependent adenoviral
vectors and their applications
for gene therapy.
0:28
Adenoviruses are among
the most commonly
used vectors in human
clinical trials.
By 2012, over 420 clinical
trials were initiated
using adenoviral vectors
with the majority being
for cancer gene therapy.
0:46
Several vectors have been
investigated for gene therapy.
And so far, no single vector
system has yet emerged as clearly
superior to the others
for all applications.
This slide shows an overview
of the strengths and weaknesses
of adenoviral vectors compared to
the most commonly used vectors,
including retroviral
and lentiviral vectors,
AAV vectors and naked plasmid DNA.
1:12
Retroviral vectors can only
transduce dividing cells, requiring
a natural breakdown of the
nuclear membrane that occurs
during cell division,
to enter the nucleus.
Therefore, actively dividing
cells, such as hematopoietic stem
cells are excellent targets for
retroviral vector mediated gene
therapy, while tissues such as the
brain, eye, lung and adult liver
are not amenable for
in vivo gene delivery.
Following the infection
of target cells,
retroviral genome integrates
into the hostage genome,
particularly in sites closer to
transcriptionally active genes,
including proto-oncogenes.
Although integration provides the
potential for long term transgene
expression, because the
integrated genome is maintained
in the progenial
transduced cells, it also
increases the risks
of cancer formation
through insertional mutagenesis
or insertional activation
of proximal genes as it was observed
in the clinical gene therapy trial
for X-linked severe
combined immunodeficiency.