Expanding the genetic editing tool kit: ZFNs, TALENs, and CRISPR-Cas9

RM Gupta, K Musunuru - The Journal of clinical investigation, 2014‏ - jci.org
The past decade has been one of rapid innovation in genome-editing technology. The
opportunity now exists for investigators to manipulate virtually any gene in a diverse range of …

[HTML][HTML] Origins of programmable nucleases for genome engineering

S Chandrasegaran, D Carroll - Journal of molecular biology, 2016‏ - Elsevier
Genome engineering with programmable nucleases depends on cellular responses to a
targeted double-strand break (DSB). The first truly targetable reagents were the zinc finger …

Introducing precise genetic modifications into human 3PN embryos by CRISPR/Cas-mediated genome editing

X Kang, W He, Y Huang, Q Yu, Y Chen, X Gao… - Journal of assisted …, 2016‏ - Springer
Purpose As a powerful technology for genome engineering, the CRISPR/Cas system has
been successfully applied to modify the genomes of various species. The purpose of this …

CRISPR-based functional genomics for neurological disease

M Kampmann - Nature Reviews Neurology, 2020‏ - nature.com
Neurodegenerative, neurodevelopmental and neuropsychiatric disorders are among the
greatest public health challenges, as many lack disease-modifying treatments. A major …

Applications of CRISPR–Cas systems in neuroscience

M Heidenreich, F Zhang - Nature Reviews Neuroscience, 2016‏ - nature.com
Genome-editing tools, and in particular those based on CRISPR–Cas (clustered regularly
interspaced short palindromic repeat (CRISPR)–CRISPR-associated protein) systems, are …

Autism-like behaviours and germline transmission in transgenic monkeys overexpressing MeCP2

Z Liu, X Li, JT Zhang, YJ Cai, TL Cheng, C Cheng… - Nature, 2016‏ - nature.com
Abstract Methyl-CpG binding protein 2 (MeCP2) has crucial roles in transcriptional
regulation and microRNA processing,,,. Mutations in the MECP2 gene are found in 90% of …

Streptococcus thermophilus CRISPR-Cas9 systems enable specific editing of the human genome

M Müller, CM Lee, G Gasiunas, TH Davis, TJ Cradick… - Molecular Therapy, 2016‏ - cell.com
RNA-guided nucleases (RGNs) based on the type II CRISPR-Cas9 system of Streptococcus
pyogenes (Sp) have been widely used for genome editing in experimental models …

Functional disruption of the dystrophin gene in rhesus monkey using CRISPR/Cas9

Y Chen, Y Zheng, Y Kang, W Yang, Y Niu… - Human molecular …, 2015‏ - academic.oup.com
CRISPR/Cas9 has been used to genetically modify genomes in a variety of species,
including non-human primates. Unfortunately, this new technology does cause mosaic …

[HTML][HTML] Brains, genes, and primates

JCI Belmonte, EM Callaway, SJ Caddick, P Churchland… - Neuron, 2015‏ - cell.com
One of the great strengths of the mouse model is the wide array of genetic tools that have
been developed. Striking examples include methods for directed modification of the …

[HTML][HTML] Modeling Rett syndrome using TALEN-edited MECP2 mutant cynomolgus monkeys

Y Chen, J Yu, Y Niu, D Qin, H Liu, G Li, Y Hu, J Wang… - Cell, 2017‏ - cell.com
Gene-editing technologies have made it feasible to create nonhuman primate models for
human genetic disorders. Here, we report detailed genotypes and phenotypes of TALEN …