Gene editing with 'pencil'rather than 'scissors' in human pluripotent stem cells
JC Park, MJ Park, SY Lee, D Kim, KT Kim… - Stem cell research & …, 2023 - Springer
Owing to the advances in genome editing technologies, research on human pluripotent stem
cells (hPSCs) have recently undergone breakthroughs that enable precise alteration of …
cells (hPSCs) have recently undergone breakthroughs that enable precise alteration of …
Clinical exome analysis and targeted gene repair of the c.1354dupT variant in iPSC lines from patients with PROM1-related retinopathies exhibiting diverse …
K Puertas-Neyra, RM Coco-Martin… - Stem Cell Research & …, 2024 - Springer
Background Inherited retinal dystrophies (IRD) are one of the main causes of incurable
blindness worldwide. IRD are caused by mutations in genes that encode essential proteins …
blindness worldwide. IRD are caused by mutations in genes that encode essential proteins …
Correction of Griscelli Syndrome Type 2 causing mutations in the RAB27A gene with CRISPR/Cas9
Background/aimGriscelli Syndrome Type 2 (GS-2) is a rare, inherited immune deficiency
caused by a mutation in the RAB27A gene. Current treatment consists of hematopoietic stem …
caused by a mutation in the RAB27A gene. Current treatment consists of hematopoietic stem …
Functional CRISPR repair of induced pluripotent stem cells from patients with Noonan syndrome-associated cardiac hypertrophy
L Roos - 2024 - ediss.uni-goettingen.de
Noonan syndrome (NS) is a multisystemic disorder and characterised by its vast clinical
variety including craniofacial dysmorphism, short stature and congenital heart defects …
variety including craniofacial dysmorphism, short stature and congenital heart defects …