BME 80H Lecture Notes - Lecture 2: Lipoprotein Lipase Deficiency, Alipogene Tiparvovec, Genentech

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2nd lecture
D. Molecular Genetics (Handout = "Molecular Genetics") link 1953-
1976, Expanding the Boundaries
-see attachment labeled handout 2
E. Modern Genetics: DNA technologies link 1977-present, Dawn of
Biotech
The advent of recombinant DNA technology and other molecular
techniques paved the way for huge advances in biomedicine and
agriculture.
1. Gene product production, making proteins from cloned
genes (table 14.1)
- first human protein produced in 1977 - human somatostatin
produced in bacteria (with company Genentech)
2. DNA and identity (DNA Forensics Information in links
lists for lecture 1- dnai)
-examining a set of DNA fragment that are unique to the
individual -DNA fingerprinting
-southern blotting and PCR are associated
-forensics, relatedness, victim identification
3. Correction of genetic defects by replacing the mutant
gene with a good copy (gene therapy) links lists for lectures 1
and 13
-first gene therapy added there p53 gene into the treatment of
cancer - called Gendicine, its the first licensed gene therapy and
it was licensed in 2003 in China only
-still only in clinical trial in US
-p53 is recognizing mutation and sending messages to repair it,
if it is too bad they send a signal for apoptosis (cell suicide)
-Glybera was licensed in 2012 in UK
-treats lipoprotein lipase deficiency (kids)
find more resources at oneclass.com
find more resources at oneclass.com
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