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Printable Handouts
Navigable Slide Index
- Introduction
- Talk outline
- Genetic therapy: definition
- Genetic therapy: goal
- Requirements for successful gene therapy
- Gene therapy medicinal products
- Viral vector systems (1)
- Advantages of retrovirus vectors
- Gene transfer using retroviral vectors
- Blood-forming stem cells as targets
- Goals of gene transfer technology using stem cells
- Disadvantages of blood stem cells as targets
- Historical perspectives
- Application in disorders of hematopoiesis
- Viral vector systems (2)
- Typical gene transfer protocol
- Severe Combined Immunodeficiency (SCID) (1)
- Severe Combined Immunodeficiency (SCID) (2)
- SCID/CID
- X-linked SCID (SCID-X1)
- Vector and protocol used in Paris and London
- Gene correction of X-SCID: French trial
- Gene correction of X-SCID: British trial
- CD3+ T lymphocytes after gc gene transfer
- Characteristics of transduced T cells
- TCRVb diversity
- ADA-SCID (1)
- ADA-SCID (2)
- Toxicity of ADA substrates
- ADA-SCID: current treatments
- Survival following HLA-mismatched transplantation
- Gene correction of ADA-SCID (1)
- Gene correction of ADA-SCID (2)
- Chronic granulomatous disease: CGD (1)
- Chronic granulomatous disease: CGD (2)
- Chronic granulomatous disease: trials
- Chronic granulomatous disease: results (1)
- Chronic granulomatous disease: results (2)
- Chronic granulomatous disease: infections
- CGD: frequency of mappable RIS
- Childhood cerebral adrenoleukodystrophy (CCALD)
- CCALD: cause, presentation, and treatment
- CCALD: a monogenic, brain demyelinating disease
- Rationale for gene transfer in CCALD
- Supporting data for gene transfer in CCALD
- CCALD: evidence of disease stabilization (1)
- CCALD: long term reconstitution
- Promising clinical data (CCALD)
- CCALD: evidence of disease stabilization (2)
- CCALD: excellent long term safety data
- Wiskott-Aldrich Syndrome (WAS) (1)
- Wiskott-Aldrich Syndrome (WAS) (2)
- WAS and gene therapy (trial in Italy)
- WAS and gene therapy (trial in Germany)
- Hematopoietic stem cell gene transfer (WAS) (1)
- Hematopoietic stem cell gene transfer (WAS) (2)
- Insertional mutagenesis
- Leukemia in X-linked SCID post gene transfer
- Clonal expansion: early detection by LAM-PCR
- Adverse event associated with gene therapy
- Retrovirus insertions near transcription start sites
- Retroviral insertion near oncogenes (leukemia)
- Safer vector design for gene therapy
- Gene transfer using a self-inactivating retrovirus
- Overall summary
- Prospective: new approaches of importance
- Thank you
Topics Covered
- Definition of gene therapy
- Requirements for successful gene therapy
- Vector systems with application for blood diseases
- Experience in clinical trials
- Insertional mutagenesis
- Prospects and new approaches
Links
Series:
Categories:
Therapeutic Areas:
Talk Citation
Williams, D.A. (2015, March 18). Gene transfer and gene therapy [Video file]. In The Biomedical & Life Sciences Collection, Henry Stewart Talks. Retrieved December 12, 2024, from https://doi.org/10.69645/XUET2684.Export Citation (RIS)
Publication History
Financial Disclosures
- Dr. David A. Williams has not informed HSTalks of any commercial/financial relationship that it is appropriate to disclose.
A selection of talks on Haematology
Transcript
Please wait while the transcript is being prepared...
0:00
This is Dave Williams,
I'm chief of hematology/oncology
at Boston Children's Hospital
and Dana Farber Cancer Institute.
And I'm going to be
talking today about gene
transfer and gene therapy.
0:14
So what I would like
to cover quickly today
is outlined in the first slide.
We'll talk a little bit about
the definition of gene therapy,
the requirements for
success of gene therapy,
in particular focusing on blood
diseases as a model system,
talk just briefly about applicable
vector systems that can be used
in blood diseases including
gamma retroviruses,
lentiviruses, foamy
and alpha retroviruses,
then briefly review experience
from clinical trials,
including the adenosine
deaminase or ADA-SCID
trial, the X-SCID trial, CGD, WAS,
CCALD trial, and beta-thalassemia,
and finally, talk about
insertional mutagenesis
as it relates to these
clinical trials and experience
with leukemias,
and end with a couple slides on
prospects and new approaches,
including new cell
targets and gene editing.
1:11
OK, so the first slide is just
a general introduction to gene
therapy by way of definition.
So we think of gene
therapy as introducing
new genetic material into
the cells of an organism
for therapeutic purposes.
There's essentially two broad
concepts or types: germline,
in which the DNA is
introduced in the germ cells
and therefore the new material
can be passed into the gene pool,
and somatic, in which the
gene sequences are introduced
into specialized somatic
cells, and therefore
the genetic material
is really limited
to the individual recipient.
And of course, the former germline
gene therapy is not practiced;
in fact is banned in most countries,
and the latter is what
we'll be focusing on today.