Gene Editing

Question 1

Surface marker for multi potent HSC

Answer 1

CD34

Question 2

Gene therapy clinical trials exist for what PIDs

Answer 2

ADA SCID
X linked SCID
Artemis SCID
LAD
CGD
Wiskott Aldrich

Question 3

What virus is used for gene therapy with lower predilection for gene integration near transcription start sites

Answer 3

Lentivirus

Question 4

Gene therapy uses programmable
Nucleases to help make it safer against leukemic transformation . Which ones?

Answer 4

Zinc fingers
TALENS (transcription activator like effector nucleases)
CRISPR/Cas 9 (clustered regularly interspaced short palindromic repeats)

Question 5

This cell expressed CD40L after it is activated

Answer 5

T cell

Question 6

Zinc fingers and TALENS binding to DNA causes dimerizarion of these nucleases

Answer 6

Fok1

Question 7

AID deficiency

Answer 7

AR hyper IgM

Question 8

%monogenic causes for PID

Answer 8

40%

Question 9

Girl with turner syndrome and no B cells . Ddx includes all except :
AID deficiency, CVID, XLA, BLNK def

Answer 9

AID def typically has normal B cells

X linked diseases can be revealed in. A girl with turner syndrome!!!