CRISPR-Cas9 genome editing articles from across Nature Portfolio

CRISPR-Cas9 genome editing exploits the CRISPR-Cas system to modify a genome in a targeted manner. Guided by RNA, the Cas9 endonuclease breaks DNA at a target sequence. Imprecise repair of the double strand break can result in insertion or deletion mutations, while repair pathways can be engineered to introduce specific point mutations or insertions.

Featured

News & Views | 19 May 2023

Gene correction for sickle cell disease hits its prime
- Sébastien Levesque
- & Daniel E. Bauer
Nature Biomedical Engineering 7, 605-606

Latest Research and Reviews

Research
19 May 2023 | Open Access

Generation of CRISPR-Cas9-mediated knockin mutant models in mice and MEFs for studies of polymorphism in clock genes
- Kwangjun Lee
- & Choogon Lee
Scientific Reports 13, 8109
Research | 15 May 2023

Deep learning models to predict the editing efficiencies and outcomes of diverse base editors

The best base editor for specific applications is predicted with a deep learning model.
- Nahye Kim
- , Sungchul Choi
- & Hyongbum Henry Kim
Nature Biotechnology, 1-14
Research
27 April 2023 | Open Access

HMGN1 enhances CRISPR-directed dual-function A-to-G and C-to-G base editing

Limited work has been done on concurrent C-to-G and A-to-G base editing. Here the authors test how a number of chromatin-associated factors affect base editing and show that HMGN1 enhanced the efficiency; by fusing HMGN1 to GBE and ABE they develop a CRISPR-based dual-function A-to-G and C-to-G base editor (GGBE).
- Chao Yang
- , Zhenzhen Ma
- & Xueli Zhang
Nature Communications 14, 2430
Research
19 April 2023 | Open Access

Direct correction of haemoglobin E β-thalassaemia using base editors

The authors demonstrate efficient and direct correction of the DNA mutation causing Haemoglobin E β-thalassaemia with CRISPR Cas9 base editors. The work includes profiling of off-target effects using deep neural networks.
- Mohsin Badat
- , Ayesha Ejaz
- & James O. J. Davies
Nature Communications 14, 2238
Research
19 April 2023 | Open Access

PAM-flexible Cas9-mediated base editing of a hemophilia B mutation in induced pluripotent stem cells

Hiramoto et al. develop a base-editing approach using SpCas9-NG, an engineered Cas9 with broad PAM flexibility, to correct a causative mutation in hemophilia B. Their approach is used to repair the point mutation in patient-derived iPSCs and restore coagulation factor IX expression in HEK293 cells and knock-in mice.
- Takafumi Hiramoto
- , Yuji Kashiwakura
- & Tsukasa Ohmori
Communications Medicine 3, 56
Protocols | 17 April 2023

Large-scale F0 CRISPR screens in vivo using MIC-Drop

This protocol for Multiplexed Intermixed CRISPR Droplets uses microfluidics to create droplets containing Cas9, multiplexed single-guide RNAs and corresponding DNA barcodes, allowing large-scale genetic screens to be performed in F0 zebrafish.
- Saba Parvez
- , Zachary J. Brandt
- & Randall T. Peterson
Nature Protocols, 1-25

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News and Comment

Research Highlights | 25 May 2023

Peptide-mediated CRISPR engineering of cells

An article in Nature Biomedical Engineering reports a simple and hardware-independent peptide-mediated delivery method for the CRISPR-mediated engineering of T cells.
- Nesma El-Sayed Ibrahim
Nature Reviews Bioengineering, 1
News & Views | 19 May 2023

Gene correction for sickle cell disease hits its prime

Prime editing can efficiently rewrite the genetic mutation causing sickle cell disease, in haematopoietic stem cells from patients.
- Sébastien Levesque
- & Daniel E. Bauer
Nature Biomedical Engineering 7, 605-606
News | 16 May 2023

Deadly mushroom poison might now have an antidote — with help from CRISPR

Gene-editing technique might have finally cracked the mystery of how death cap mushrooms kill.
- Saima Sidik
Nature
Editorial | 25 April 2023

The gene-therapy revolution risks stalling if we don’t talk about drug pricing

Regulation and new intellectual property laws are needed to reduce the cost of gene-editing treatments and fulfil their promise to improve human health.

Nature 616, 629-630
Comments & Opinion
29 March 2023 | Open Access

A gene drive is a gene drive: the debate over lumping or splitting definitions

We address a controversy over use of the term “gene drive” to include both natural and synthetic genetic elements that promote their own transmission within a population, arguing that this broad definition is both practical and has advantages for risk analysis.
- Stephanie L. James
- , David A. O’Brochta
- & Omar S. Akbari
Nature Communications 14, 1749
News | 10 March 2023

Why CRISPR babies are still too risky — embryo studies highlight challenges

While society grapples with the social and ethical implications of heritable genome editing, technical obstacles still abound.
- Heidi Ledford
Nature 615, 568-569

All News & Comment

CRISPR-Cas9 genome editing articles from across Nature Portfolio

Featured

Gene correction for sickle cell disease hits its prime

Latest Research and Reviews

Generation of CRISPR-Cas9-mediated knockin mutant models in mice and MEFs for studies of polymorphism in clock genes

Deep learning models to predict the editing efficiencies and outcomes of diverse base editors

HMGN1 enhances CRISPR-directed dual-function A-to-G and C-to-G base editing

Direct correction of haemoglobin E β-thalassaemia using base editors

PAM-flexible Cas9-mediated base editing of a hemophilia B mutation in induced pluripotent stem cells

Large-scale F0 CRISPR screens in vivo using MIC-Drop

News and Comment

Peptide-mediated CRISPR engineering of cells

Gene correction for sickle cell disease hits its prime

Deadly mushroom poison might now have an antidote — with help from CRISPR

The gene-therapy revolution risks stalling if we don’t talk about drug pricing

A gene drive is a gene drive: the debate over lumping or splitting definitions

Why CRISPR babies are still too risky — embryo studies highlight challenges

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