Delivery of CRISPR-Cas tools for in vivo genome editing therapy: Trends and challenges

EA Taha, J Lee, A Hotta - Journal of Controlled Release, 2022 - Elsevier
The discovery of clustered regularly interspaced short palindromic repeats (CRISPR)
genome editing technology opened the door to provide a versatile approach for treating …

CRISPR-based genome editing through the lens of DNA repair

TS Nambiar, L Baudrier, P Billon, A Ciccia - Molecular cell, 2022 - cell.com
Genome editing technologies operate by inducing site-specific DNA perturbations that are
resolved by cellular DNA repair pathways. Products of genome editors include DNA breaks …

Advances in genome editing through control of DNA repair pathways

CD Yeh, CD Richardson, JE Corn - Nature cell biology, 2019 - nature.com
Eukaryotic cells deploy overlapping repair pathways to resolve DNA damage.
Advancements in genome editing take advantage of these pathways to produce permanent …

Methods favoring homology-directed repair choice in response to CRISPR/Cas9 induced-double strand breaks

H Yang, S Ren, S Yu, H Pan, T Li, S Ge… - International journal of …, 2020 - mdpi.com
Precise gene editing is—or will soon be—in clinical use for several diseases, and more
applications are under development. The programmable nuclease Cas9, directed by a …

Methodologies for improving HDR efficiency

M Liu, S Rehman, X Tang, K Gu, Q Fan, D Chen… - Frontiers in …, 2019 - frontiersin.org
Clustered regularly interspaced short palindromic repeats (CRISPR)-associated protein 9
(Cas9) is a precise genome manipulating technology that can be programmed to induce …

Easi-CRISPR: a robust method for one-step generation of mice carrying conditional and insertion alleles using long ssDNA donors and CRISPR ribonucleoproteins

RM Quadros, H Miura, DW Harms, H Akatsuka, T Sato… - Genome biology, 2017 - Springer
Background Conditional knockout mice and transgenic mice expressing recombinases,
reporters, and inducible transcriptional activators are key for many genetic studies and …

Efficient generation of targeted large insertions by microinjection into two-cell-stage mouse embryos

B Gu, E Posfai, J Rossant - Nature biotechnology, 2018 - nature.com
Rapid, efficient generation of knock-in mice with targeted large insertions remains a major
hurdle in mouse genetics. Here, we describe two-cell homologous recombination (2C-HR) …

Dynamics and competition of CRISPR–Cas9 ribonucleoproteins and AAV donor-mediated NHEJ, MMEJ and HDR editing

YW Fu, XY Dai, WT Wang, ZX Yang… - Nucleic acids …, 2021 - academic.oup.com
Investigations of CRISPR gene knockout editing profiles have contributed to enhanced
precision of editing outcomes. However, for homology-directed repair (HDR) in particular …

Hematopoietic stem cell gene therapy: progress and lessons learned

RA Morgan, D Gray, A Lomova, DB Kohn - Cell stem cell, 2017 - cell.com
The use of allogeneic hematopoietic stem cells (HSCs) to treat genetic blood cell diseases
has become a clinical standard but is limited by the availability of suitable matched donors …

CtIP fusion to Cas9 enhances transgene integration by homology-dependent repair

M Charpentier, AHY Khedher, S Menoret… - Nature …, 2018 - nature.com
In genome editing with CRISPR–Cas9, transgene integration often remains challenging.
Here, we present an approach for increasing the efficiency of transgene integration by …