Tissue Exam 3 Tools in TE

gene therapy

the introduction of genetic material (DNA or RNA) into existing cells

goal of gene therapy

to restore the function and inhibit the function of faulty or defective gene

insert, remove, change, or replace

gene editing is used to

goal of gene editing

to directly edit or change DNA to its correct code

engineered nucleases

gene editing uses ______ to create site specific double strand breaks

NHEJ or homologous recombination

induced double strand breaks are repaired through ____

clustered regularly interspaced short palindromic repeats

CRISPR stands for

somatic cell and germ line

types of gene therapy/editing

reproductive

somatic cells are any cell other than ___ cells

embryonic

germ cells are ____ cells with potential to develop into gamete

not heritable

somatic cells are ____

heritable

germ line cells are ____

homology directed repair

which double strand break repair pathway is necessary to introduce donor/template DNA into the genome?

ex vivo or in vivo

methods of gene delivery

autologous cells

these are collected from the patient, genetically manipulated, and returned to host

adenosine deaminase (ADA)

1st gene therapy to correct deficiency of enzyme

in vivo gene therapy/editing

direct delivery of therapeutic genes into target cells in the patients body