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Brief about Gene Therapy: Review

Gaurav Singh

Abstract


The ability to make site-specific modifications to the human genome has been a goal in medicine since
the discovery of the gene as the basic unit of heredity. As a result, gene therapy is defined as the ability
to change one's genetic condition by correcting altered (mutated) genes or making site-specific
modifications that target therapeutic treatment. Developments in genetics and bioengineering enabled
the use of vectors to transport extra-chromosomal material to target cells, making this treatment
possible. The use of delivery vectors, which are mostly plasmids, is one of the main part of this
technique. Viruses are studied more frequently because of their prowess at invading cells and inserting
genetic material. However, heightened immunological responses and genetic alteration, particularly in
germ line cells, are major concerns. In vivo investigations in in somatic cells yielded positive results in
clinical trials using approved methods. Recent biotechnological breakthroughs include induced
pluripotent stem cells in liver disease patients, chimeric antigen receptor T-cell immunotherapy, and
CRISPR/Cas9 genome editing.


Keywords


Gene therapy, Genetic Vectors, CRISPR-Cas9, Genetic therapy, Gene transfer

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References


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