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Printable Handouts
Navigable Slide Index
- Introduction
- Factors required for successful CGT transgene delivery
- Major milestones in Gene therapies
- Schematic of ex vivo and in vivo strategies
- Where/How do you want to apply CGT? (1)
- Where/How do you want to apply CGT? (2)
- Where/How do you want to apply CGT? (3)
- What is an ATMP?
- Why do we need CGT and ATMPs?
- What are the indications addressed by and vectors used in CGT clinical trials?
- Clinical phases and gene types transferred in CGT clinical trials
- Landscape of early clinical CGT (non-oncology)
- Vector development: using the right vector for the right target/modality
- CGT gene delivery approaches: non viral
- CGT gene delivery approaches: current toolbox of viruses
- CGT platforms & delivery technologies
- Gene editing technologies for the treatment of human diseases
- CGT PD&S: vector production atlarge scale
- CGT PD&S: potential variability and control of transient production
- CGT PD&S: innovation in lentiviral vector production
- The use of viral vectors in gene therapy
- AAV technology: from virus to vector (1)
- AAV technology: from virus to vector (2)
- Setting up the system
- Serotype specificity for disease targets
- Challenges of vector-cell interactions
- AAV Engineering
- Engineering strategies for AAV vectors
- Surface engineering lentiviral vectors
- Avoiding immune responses in gene therapy
- The role of small molecules in cell and gene therapy
- GSK’s CGT strategy
- GSK’s CGT strategy: break through innovation therapies
Topics Covered
- Major milestones in gene therapies
- Ex vivo and in vivo strategies of cell and gene therapy (CGT)
- The importance of CGT
- CGT gene delivery via non-viral approaches
- CGT platforms and delivery technologies
- The use of viral vectors in gene therapy
- Adeno-associated virus vector engineering
- Surface engineering lentiviral vectors
- The cell and gene therapy strategy of GSK
Links
Series:
Categories:
External Links
Talk Citation
Athanasopoulos, T. (2022, February 23). Recent advances in the development of gene delivery technologies [Video file]. In The Biomedical & Life Sciences Collection, Henry Stewart Talks. Retrieved December 10, 2024, from https://doi.org/10.69645/KIAD4459.Export Citation (RIS)
Publication History
Financial Disclosures
- Dr. Takis Athanasopoulos has not informed HSTalks of any commercial/financial relationship that it is appropriate to disclose.
Other Talks in the Series: Periodic Reports: Advances in Clinical Interventions and Research Platforms
Transcript
Please wait while the transcript is being prepared...
0:00
Hello to all.
My name is Dr. Takis Athanasopoulos
from GlaxoSmithKline.
I'm the Acting Head
of Patient Operations
in Cell and Gene Therapy
at GSK in Stevenage, UK.
The focus of our talk
today will be on the
recent advances in
the development
of gene delivery technologies.
It is an important
goal in the research
of gene delivery systems to develop
clinically relevant vectors
that we can use to combat
a lot of diseases.
We have quite a few
HIV, AIDS, cancer, Alzheimer's
and monogenic diseases.
DNA or RNA-based viral
vector systems utilise
these viral or non-viral
vector technologies
to deliver genetic
materials to host cells.
It is an efficient way to
deliver the genetic material
and we will be capturing some of these
recent technologies as part of our talk.
1:01
Depicted here are some of the
critical factors that are
required for a successful
cell and gene therapy.
In terms of delivery,
a viral or non-viral
vector has lots
of cell structures and defences
to beat during their uptake.
They have to utilise
their cellular receptors,
or co-receptors,
but in order to work, it's
trafficked towards the cell.
There it will interact
with multiple organelles,
depending on whether it
is a DNA or RNA vector.
The eukaryotes include
animals, plants and fungi,
and we have multiple organelles
with various functions.
For example,
the nucleolus that
makes the ribosomes,
the mitochondria which are the
energy producers of the cell,
the Golgi complex, an apparatus
that processes proteins and lipids
and are useful for the
secretion of the cell,
and microtubules that are for
structural movement and cell division.
The rough and the smooth endoplasmic
reticulum for lipid synthesis and metabolism,
and lysosomes and
various other vesicles.
Of importance to all these
critical structures,
and from a gene
transfer perspective,
is the uptake of the vector
by appropriate cells,
but also the subsequent
steps, i.e., the vector entry
into the nucleus if it is a DNA vector,
or the cytoplasm if it is an RNA vector.
Of paramount importance,
from a gene transfer
perspective,
is the evasion of
host immune defences.
One of the factors required for
successful cell and gene
therapy transgene delivery
is that the vector has to evade
the host immune defences.
These evasions are
of multiple natures.
It can be an innate
response with
subsequent release of
pro-inflammatory cytokines,
recruitment of
neutrophils, macrophages,
dendritic cells or
other leukocytes,
and activation and maturation
of antigen-presenting cells.
It can also involve adaptive priming
through dendritic cell activation
or adaptive responses.
All of these are very important.
From a cell and gene transfer
delivery perspective,
we want to abdicate or eliminate
any unwanted immune responses.