gene therapy Flashcards
which therapy is inhereted to the other generation which is not ?
somatic
girm line gene therapy
which is targeting of gerem cells
for patients with genetic diseases ;what is the mosst common approach for them?
it is to construct a viral vector containing the human complamantry DNA
what is the gene therapy stand for
CRISPER-CAS9
clustered
regulary
interspaced
short
palindromic
repeats
associated protein 9
CRISPR-Cas 9 adopted from ?
defens mechanism against the virus of bacteria
what is cas9 and what is for ?
an enzme use guid RNA leading to cut target DNA sequences
and colud add in repairing system for customize DNA
double strand break by ?
broviding a donor dna in trans
transgenic DNA can be created
if the DNA donor what hhappen ?
host cell will repair the break strand which will cuz distrupting the opr reading frame of gene
the research is a proof-of-concept that///
monogeneic forms of diabetes can be cured by
correcting the diabetes-cusing mutation in stem cells drived from patient’s own cells
which artical of diabetes
wolframs syndrome treatment
crisp-case 9 to reduce alpah globin
beta thalassemia tretment
SMA treatment
that the study proof-of-principle a new strategy to therapeutically intervene in SMA and other RNA splicing-related diseases
CRISPR/CAS9 technology offers a
to explore the genetic causes of diseases
evaluate therapeutic stratiges
risks of gene therapy?
adverse response to the vectore-disease combination
insertional mutagenesis causing malignancy
insertional inactivation of an essential gene
