Gene therapy (also called human gene transfer) is a medical field
which focuses on the utilization of the therapeutic delivery of
nucleic acids into a patient's cells as a drug to treat disease. The
first attempt at modifying human DNA was performed in 1980 by Martin
Cline, but the first successful nuclear gene transfer in humans,
approved by the National Institutes of Health, was performed in May
1989. The first therapeutic use of gene transfer as well as the first
direct insertion of human DNA into the nuclear genome was performed by
French Anderson in a trial starting in September 1990. It is thought
to be able to cure many genetic disorders or treat them over
time.Between 1989 and December 2018, over 2,900 clinical trials were
conducted, with more than half of them in phase I. As of 2017, Spark
Therapeutics' Luxturna (RPE65 mutation-induced blindness) and
Novartis' Kymriah (Chimeric antigen receptor T cell therapy) are the
FDA's first approved gene therapies to enter the market. Since that
time, drugs such as Novartis' Zolgensma and Alnylam's Patisiran have
also received FDA approval, in addition to other companies' gene
therapy drugs. Most of these approaches utilize adeno-associated
viruses (AAVs) and lentiviruses for performing gene insertions, in
vivo and ex vivo, respectively. ASO / siRNA approaches such as those
conducted by Alnylam and Ionis Pharmaceuticals require non-viral
delivery systems, and utilize alternative mechanisms for trafficking
to liver cells by way of GalNAc transporters.The concept of gene
therapy is to fix a genetic problem at its source. If, for instance,
in an (usually recessively) inherited disease a mutation in a certain
gene results in the production of a dysfunctional protein, gene
therapy could be used to deliver a copy of this gene that does not
contain the deleterious mutation, and thereby produces a functional
protein. This strategy is referred to as gene replacement therapy and
is employed to treat inherited retinal diseases.
which focuses on the utilization of the therapeutic delivery of
nucleic acids into a patient's cells as a drug to treat disease. The
first attempt at modifying human DNA was performed in 1980 by Martin
Cline, but the first successful nuclear gene transfer in humans,
approved by the National Institutes of Health, was performed in May
1989. The first therapeutic use of gene transfer as well as the first
direct insertion of human DNA into the nuclear genome was performed by
French Anderson in a trial starting in September 1990. It is thought
to be able to cure many genetic disorders or treat them over
time.Between 1989 and December 2018, over 2,900 clinical trials were
conducted, with more than half of them in phase I. As of 2017, Spark
Therapeutics' Luxturna (RPE65 mutation-induced blindness) and
Novartis' Kymriah (Chimeric antigen receptor T cell therapy) are the
FDA's first approved gene therapies to enter the market. Since that
time, drugs such as Novartis' Zolgensma and Alnylam's Patisiran have
also received FDA approval, in addition to other companies' gene
therapy drugs. Most of these approaches utilize adeno-associated
viruses (AAVs) and lentiviruses for performing gene insertions, in
vivo and ex vivo, respectively. ASO / siRNA approaches such as those
conducted by Alnylam and Ionis Pharmaceuticals require non-viral
delivery systems, and utilize alternative mechanisms for trafficking
to liver cells by way of GalNAc transporters.The concept of gene
therapy is to fix a genetic problem at its source. If, for instance,
in an (usually recessively) inherited disease a mutation in a certain
gene results in the production of a dysfunctional protein, gene
therapy could be used to deliver a copy of this gene that does not
contain the deleterious mutation, and thereby produces a functional
protein. This strategy is referred to as gene replacement therapy and
is employed to treat inherited retinal diseases.
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