Marina

AT

Drugs to be tested:

1. Splicing modulators.

2. Anti-oxidants

A. Vit E

B. N-acetyl-cystein

AT Treatment:

1. Bethametazon (steroid: dexamethazon) to irradiation and neural differentiation.

2. IgG

Anti-sense morpholin

1. Readthrough drug for DMD.

RALD: Ras-associated ALPS-like disorder

AAV1-locus

PiggyBac-ATM

ATM: mini-gne

electroporation

LD

Generation of iPSCs