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Bioengineering for Gene Therapy by David Schaffer #rejbio

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Molecular engineering of gene therapies for the nervous system

DNA as medicine

Historical timeline in molecular medicine development
started with small molecules (1908 start)
Protein therapies (1920s start)
gene therapies (1980s had some therapies that were somewhat effective)
Stem cells (1960s start)

More chronic health conditions are now the problems.
Chronic age related disease
alzheimers, parkinsons, etc...

Three problems of gene therapy - delivery, delivery and delivery

Adeno associated virus (5 kilobases - AAV)
60 units of CAP protein assemble to form icosahedral capsid

numerous natural variants have been isolated
non-pathogenic.

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