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Printable Handouts
Navigable Slide Index
- Introduction
- Contents
- What is gene therapy?
- Why do we need gene therapy?
- Initial days and progress of gene therapy
- Gene therapy timeline
- Basics of gene therapy - types
- Basics of gene therapy - approaches
- Suppression of gene expression
- Mechanism of gene suppression by RNAi
- Over-expression/replacement of genes
- Gene modification/editing
- Gene editing: strategies
- Gene editing: CRISPR/Cas9
- Gene editing: summary
- Gene transfer
- Methods of gene transfer
- Methods of gene transfer: physical
- Methods of gene transfer: chemical
- Methods of gene transfer: biological
- Mechanism of gene transfer: viral and non-viral
- Mechanism of gene transfer: AAV
- Mechanism of gene transfer: summary
- Hurdles in gene transfer
- Ex vivo and in vivo gene transfer (1)
- Ex vivo and in vivo gene transfer (2)
- Ex vivo gene transfer: schematic
- Therapeutic applications of gene therapy (1)
- Therapeutic applications of gene therapy (2)
- Therapeutic applications of gene therapy (3)
- Summary
- Thank you
Topics Covered
- Introduction to gene therapy
- Basic concepts of gene therapy
- Methods of gene transfer
- Ex vivo and in vivo gene transfer
- Therapeutic applications of gene therapy
Talk Citation
Khurana, A. (2023, April 30). Gene therapy: basics and therapeutic applications [Video file]. In The Biomedical & Life Sciences Collection, Henry Stewart Talks. Retrieved October 7, 2024, from https://doi.org/10.69645/OUNT7306.Export Citation (RIS)
Publication History
Financial Disclosures
- Dr. Amit Khurana has not informed HSTalks of any commercial/financial relationship that it is appropriate to disclose.
A selection of talks on Pharmaceutical Sciences
Transcript
Please wait while the transcript is being prepared...
0:00
Hello everyone. I am
Dr. Amit Khurana,
a Senior Researcher at
the Institute of Molecular
Pathobiochemistry,
Experimental Gene Therapy,
and Clinical Chemistry,
at the University Hospital
RWTH Aachen, Aachen Germany.
I have a PhD in Pharmacology
and Toxicology,
and my primary research area
is the use of novel
technologies,
including gene therapy
for the treatment of
chronic inflammation and
fibrosis driven diseases.
In this lecture,
I will introduce you to some of
the important basic concepts of
gene therapy and its
therapeutic applications.
0:43
In this lecture, I will be
covering first the
introduction to gene therapy,
followed by understanding some
of the basic concepts
of gene therapy.
Next, I will provide
the information
about the various methods
of gene transfer,
then we will study
what is ex vivo
and in vivo gene transfer in
the context of gene therapy.
Finally, we will
learn about some of
the areas of clinical
applications of gene therapy.
1:14
Now, the important
question comes.
What is gene therapy?
To define it, gene therapy
is the medical field which
focuses on the
genetic modification
of cells to produce a
therapeutic effect.
Or it involves the
treatment of disease by
repairing or reconstructing
defective genetic material.
In a nutshell, gene
therapy essentially
deals with manipulating
the genetic material
that is deoxyribonucleic
acid, DNA
by using different approaches.