GENE THERAPY

Khadija Tariq
Bs-Genetics
What is gene therapy?
• Use of genetic material to cure or prevent diseases.
• Can be done in different ways through different methods
 Replacing genes
 Inactivating genes
 Introducing new genes

• Clinical trials and ethical boundaries

History of gene therapy
• 1960s experiments on HPRT deficient cell,
confirming gene transfer
• Late 1960s experiments on papovaviruses SV40
and plyoma
• 1970s recombinant DNA technology
• 1979 clinical trials of Marin Cline
• 1981-2 use of retroviral vectors with 100% efficiency
• Banbury conference in 1983 discussing techniques
on gene therapy
• 1989-90 human clinical trial approved
Types of gene therapy
Somatic gene therapy Germ line gene therapy
• The methods in which • The methods in which the
somatic or non-reproductive germ line cells are
cells are transformed transformed
• Types
 In-vivo - the methods in which the cells are
• The affects are inheritable
transformed inside the body to the next generation
 Ex-vivo – the methods in which the cells are
transformed outside the body • The side effects are
• The affects are non- unknown
heritable • Banned in a lot of
• Drawbacks include short countries of the world.
lived affects/ repeated
therapy
Vectors
Viral vectors Non-viral vectors

• The genetic material is • The genetic material is

targeted to host cells via targeted through non-viral
genetically engineered methods such as
viruses
• Naked DNA
• Viruses used are retroviruses,
Such as plasmid injections
adenoviruses etc.
• Oligonucleotides
• Depends on the infection,
Bind to transcription factors to
host spectrum and transgene
prevent expression of a faulty gene
expression of the virus
• Lipoplexes
• Drawbacks include
Liposomal structures to protect DNA
overexpression, wide host
spectrum etc.
Applications

• Gene doping

• Genetic engineering