Gene therapy
In vivo gene editing of human hematopoietic stem and progenitor cells using envelope-engineered virus-like particles
kakameo
Cloning of constructs Amino acid sequences for VSV-G, BaEVTR31 and BaEVTR_MACA variant were codon-optimized, synthesized (ATUM or Twist) and cloned ...
FDA adds strongest warning to Sarepta gene therapy linked to 2 patient deaths
kakameo
WASHINGTON — On Friday, the Food and Drug Administration said it would add a new warning and other restrictions to ...
Site-specific DNA insertion into the human genome with engineered recombinases
kakameo
Ethics statement Our research complies with relevant ethical regulations. Experiments using hESC lines were performed under an allowance granted by ...
Design of optimized epigenetic regulators for durable gene silencing with application to PCSK9 in nonhuman primates
kakameo
Leibowitz, M. L. et al. Chromothripsis as an on-target consequence of CRISPR–Cas9 genome editing. Nat. Genet. 53, 895–905 (2021). Article ...
