Question: How to find Mendelian monogenic defects amenable to AAV trasduction gene editing?
gravatar for gogonegro
9 months ago by
gogonegro0 wrote:

Please bear with a total newbie :)

a) Where and how would I look for a list of known genetic mutations which produce problems in humans and are monogenic?

b) How would I restrict the above list to mutation that are amenable (at least in theory) to transduction of a plasmid made of GeneOfInterest+Crspr/Cas9 or similar techniques using a goven vector say an AAV, knowing that the maximum payload is say (just as an example) 4kb?

In what databases would you search to find the above information?

Thank you very much

ADD COMMENTlink written 9 months ago by gogonegro0
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