studies used to identify a target for sickle cell disease
type of modifications in ASOs that prevent enzymatic degradation
miR-128-1 acts as a switch to repress energy expenditure gene programs to promote___
when one gene influences multiple phenotypic traits
__ diseases are an example of a loss of function disease
You would do a ____-based approach if you want to understand what the chemical outcomes of your microbiome activities are?
type of LNA that works by cleaving the target RNA and is very stable
where CRISPR-cas systems were initially discovered
You would do a ____-based approach if you want to understand how your microbiome is interacting with the host and what proteins are being produced?
Verticales
___ new bacterial metabolism can create new strains that can persist in the gut, eliminate unwanted metabolism, correct metabolic deficiencies, or provide a desirable compound.
disease that is caused by mutations in CFTR chloride channel that cause mucus to accumulate in the lungs and makes the patient susceptible to infection
bacteria are____
an example of a gain of function disease
The effect of a gene mutation is dependent on the presence or absence of mutations in one or more other genes (one thing standing in front of the other).
the target for CRISPR-cas therapy in sickle cell disease is the BCL11A ___
Antisense Oligonucleotides are ___ , typically 10-20 nucleotides
drug that inhibits a small subset of tyrosine kinases (including BCR-ABL) by mimicking ATP
type of LNA that binds and blocks its target RNA with high affinity
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