A new class of medicines through DNA editing

MH Porteus - New England Journal of Medicine, 2019 - Mass Medical Soc
Clinical Genome Editing Therapeutic editing of the human genome has long been a holy
grail of genetic medicine. Different approaches are founded on a bedrock of basic laboratory …

DNA transposons and the evolution of eukaryotic genomes

C Feschotte, EJ Pritham - Annu. Rev. Genet., 2007 - annualreviews.org
Transposable elements are mobile genetic units that exhibit broad diversity in their structure
and transposition mechanisms. Transposable elements occupy a large fraction of many …

Frequency and mechanisms of LINE-1 retrotransposon insertions at CRISPR/Cas9 sites

J Tao, Q Wang, C Mendez-Dorantes, KH Burns… - Nature …, 2022 - nature.com
CRISPR/Cas9-based genome editing has revolutionized experimental molecular biology
and entered the clinical world for targeted gene therapy. Identifying DNA modifications …

Uridine depletion and chemical modification increase Cas9 mRNA activity and reduce immunogenicity without HPLC purification

S Vaidyanathan, KT Azizian, AKMA Haque… - … Therapy-Nucleic Acids, 2018 - cell.com
The Cas9/guide RNA (Cas9/gRNA) system is commonly used for genome editing. mRNA
expressing Cas9 can induce innate immune responses, reducing Cas9 expression. First …

Knock-in of large reporter genes in human cells via CRISPR/Cas9-induced homology-dependent and independent DNA repair

X He, C Tan, F Wang, Y Wang, R Zhou… - Nucleic acids …, 2016 - academic.oup.com
Abstract CRISPR/Cas9-induced site-specific DNA double-strand breaks (DSBs) can be
repaired by homology-directed repair (HDR) or non-homologous end joining (NHEJ) …

An unbiased genome-wide analysis of zinc-finger nuclease specificity

R Gabriel, A Lombardo, A Arens, JC Miller… - Nature …, 2011 - nature.com
Zinc-finger nucleases (ZFNs) allow gene editing in live cells by inducing a targeted DNA
double-strand break (DSB) at a specific genomic locus. However, strategies for …

Molecular Poltergeists: Mitochondrial DNA Copies (numts) in Sequenced Nuclear Genomes

E Hazkani-Covo, RM Zeller, W Martin - PLoS genetics, 2010 - journals.plos.org
The natural transfer of DNA from mitochondria to the nucleus generates nuclear copies of
mitochondrial DNA (numts) and is an ongoing evolutionary process, as genome sequences …

Homology-driven genome editing in hematopoietic stem and progenitor cells using ZFN mRNA and AAV6 donors

J Wang, CM Exline, JJ DeClercq, GN Llewellyn… - Nature …, 2015 - nature.com
Genome editing with targeted nucleases and DNA donor templates homologous to the
break site has proven challenging in human hematopoietic stem and progenitor cells …

Impact of the KU80 pathway on NHEJ-induced genome rearrangements in mammalian cells

J Guirouilh-Barbat, S Huck, P Bertrand, L Pirzio… - Molecular cell, 2004 - cell.com
Using a substrate measuring deletion or inversion of an I-SceI-excised fragment and both
accurate and inaccurate rejoining, we determined the impact of non-homologous end …

Directed networks reveal genomic barriers and DNA repair bypasses to lateral gene transfer among prokaryotes

O Popa, E Hazkani-Covo, G Landan, W Martin… - Genome …, 2011 - genome.cshlp.org
Lateral gene transfer (LGT) plays a major role in prokaryote evolution with only a few genes
that are resistant to it; yet the nature and magnitude of barriers to lateral transfer are still …