Gene Therapy/Gene Precision Medicine

ch. 19 p 11-12 pg. 602-604, ch. 20 pg. 8=618.622, article

human: 3.2 bill dna bp per haploid genome

precision medicine: medical care designed to optimize efficiency or therapeutic benefit for particular groups of patients, especially by using genetic or molecular profiling ex: patients w tumors that share same geneetic change get drug that targets that change, no matter what cancer.

genome 99% alike but diff by single nucleotide polymorphisms (SNPs)-important for medical treatment

SNP: site in genome where indivd. members of a species differ in single base pair.

to be considered snp: frequency of at least 1% in population-very rare variations not snps.

inherited as allelic variants in same way as alleles that produce phenotypic differences, but snps often do not produce phenotypic differences.

snp found approx. every 1000 bp.

SNPS: copying errors

gene therapy: introducing dna that encodes a functional, therapeutic gene to replace mutated gene. ex: adno-associated virus serotype 1 viral vector

adeno-associated virus: pros: doesn;t cause immune reaction. cons: holds small amount of DNA; hard to produce

Lentivirus: pros: can accomadate large genes cons: saftey concerns

Gene therapy:

ex 1: AAV. cells not sensitive to light

ex 2: gamma retrovirus