EM Handel, T Cathomen - Current gene therapy, 2011 - ingentaconnect.com
Therapeutic genome engineering is a hallmark of gene therapy but only recent technological advances have permitted the modification of complex genomes in a targeted …
R Banakar, AL Eggenberger, K Lee, DA Wright… - Scientific Reports, 2019 - nature.com
An important advantage of delivering CRISPR reagents into cells as a ribonucleoprotein (RNP) complex is the ability to edit genes without reagents being integrated into the …
CRISPR/Cas technologies have transformed our ability to manipulate genomes for research and gene-based therapy. In particular, homology-directed repair after genomic cleavage …
JB Owens, D Mauro, I Stoytchev, MS Bhakta… - Nucleic acids …, 2013 - academic.oup.com
Insertional therapies have shown great potential for combating genetic disease and safer methods would undoubtedly broaden the variety of possible illness that can be treated. A …
SH Rahman, ML Maeder, JK Joung… - Human gene …, 2011 - liebertpub.com
Zinc-finger nucleases (ZFNs) are a powerful tool that can be used to edit the human genome ad libitum. The technology has experienced remarkable development in the last few years …
L Davis, N Maizels - PloS one, 2011 - journals.plos.org
Targeted gene correction employs a site-specific DNA lesion to promote homologous recombination that eliminates mutation in a disease gene of interest. The double-strand …
G Yi, JG Choi, P Bharaj, S Abraham, Y Dang… - … Therapy-Nucleic Acids, 2014 - cell.com
CCR5 disruption by zinc finger nucleases (ZFNs) is a promising method for HIV-1 gene therapy. However, successful clinical translation of this strategy necessitates the …
KR Smith, S Chan, J Harris - Archives of medical research, 2012 - Elsevier
The latest mammalian genetic modification technology offers efficient and reliable targeting of genomic sequences, in the guise of designer genetic recombination tools. These and …