ArticleNature2025
Custom CRISPR-Cas9 PAM variants via scalable engineering and machine learning.
Abstract read
In one paragraph
Article in Nature, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 41 papers.
0numbers the graph read from it
0cells of the map it votes in
41citing papers in PubMed
–field-weighted citation impact
What it found
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The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
41 citing papers in PubMed.
- Machine-learning in optimization of CRISPR technology.Machine learning. Health · 2026Review
- CRISPR decodes the RNA regulatory network in prostate cancer: A review from mechanisms to precision therapeutics.Non-coding RNA research · 2026Review
- Technical limitations of CRISPR-Cas9 genome editing in bacteria: challenges and future directions.Archives of microbiology · 2026Review
- Base editing for precision therapeutics.Cell genomics · 2026Review
- Improved specificity and efficiency of in vivo adenine base editing therapies with hybrid guide RNAs.Nature biomedical engineering · 2026Article
- CRISPR-Cas9 and precision editing technologies linking functional genomics to clinical translation in genetic diseases.Clinical and translational medicine · 2026Review
- Adaptive model-guided protein evolution with sparse data optimizes compact eukaryotic genome editors.Nature biotechnology · 2026Article
- Compact 9dBEs Enable Efficient and Precise Genome Editing in Mammalian Cells and In Vivo.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026Article
- On-target and off-target activities of CRISPR therapeutics across scales.Trends in biotechnology · 2026Review
- Prime Editing Mediated Generation and Correction of the mdx5cv Mutation Restores Dystrophin Expression in Myoblasts.International journal of molecular sciences · 2026Article
- Article
- Adenine base editor for knockout of proteins: A practical guide from design to analysis with updated MultiEditRbatch.Molecular therapy. Nucleic acids · 2026Article
- dSaCas9 enables enhanced transcriptional activation in Nicotiana benthamiana compared to its dSpCas9 ortholog.BMC plant biology · 2026Article
- Generalist biological artificial intelligence in modeling the language of life.Nature biotechnology · 2026Review
- SynFit: Synergistic Contrastive Learning for Multi-Objective Protein Fitness Prediction and Optimization.bioRxiv : the preprint server for biology · 2026Article
- AI-guided redesign of laboratory-evolved reverse transcriptases enhances prime editing.Nature biotechnology · 2026Article
- Towards precision medicine for brain arteriovenous malformations.The Journal of clinical investigation · 2026Review
- PAM-flexible SpCas9 variants expand the targeting scope for porcine genome editing and cellular disease modeling.BMC biotechnology · 2026Article
- Engineering of a bespoke CRISPR base editor to treat a severe vascular disease.Nature biomedical engineering · 2026Article
- Treatment of a severe vascular disease using a bespoke CRISPR-Cas9 base editor in mice.Nature biomedical engineering · 2026Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
25 authors.
Rachel A SilversteinPhD Program in Biological and Biomedical Sciences, Harvard Medical School, Boston, MA, USA.
Nahye Kim *Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.
Ann-Sophie Kroell *Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.ORCID 0000-0001-9986-0737
Russell T WaltonCenter for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.ORCID 0000-0002-0546-474X
Justin DelanoDepartment of Biomedical Informatics, Harvard Medical School, Boston, MA, USA.ORCID 0000-0002-7988-5352
Rossano M ButcherOcular Genomics Institute, Massachusetts Eye and Ear, Boston, MA, USA.
Martin PacesaLaboratory of Protein Design and Immunoengineering, École Polytechnique Fédérale de Lausanne and Swiss Institute of Bioinformatics, Lausanne, Switzerland.ORCID 0000-0003-1560-5769
Blaire K SmithCenter for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.
Kathleen A ChristieCenter for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.
Leillani L HaCenter for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.ORCID 0000-0003-4478-660X
Ronald J MeisCELLSCRIPT, Madison, WI, USA.
Aaron B ClarkCELLSCRIPT, Madison, WI, USA.
Aviv D SpinnerPhD Program in Biological and Biomedical Sciences, Harvard Medical School, Boston, MA, USA.
Cicera R LazzarottoDepartment of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.
Yichao LiDepartment of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.ORCID 0000-0001-5791-096X
Azusa MatsubaraDepartment of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.ORCID 0009-0007-1637-6386
Elizabeth O UrbinaDepartment of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.
Gary A DahlCELLSCRIPT, Madison, WI, USA.
Bruno E CorreiaLaboratory of Protein Design and Immunoengineering, École Polytechnique Fédérale de Lausanne and Swiss Institute of Bioinformatics, Lausanne, Switzerland.ORCID 0000-0002-7377-8636
Debora S MarksBroad Institute of Harvard and MIT, Cambridge, MA, USA.
Shengdar Q TsaiDepartment of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.ORCID 0000-0001-9161-3993
Luca PinelloDepartment of Pathology, Harvard Medical School, Boston, MA, USA.ORCID 0000-0003-1109-3823
Suk See De RavinLaboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, USA.ORCID 0000-0002-9800-774X
Qin LiuOcular Genomics Institute, Massachusetts Eye and Ear, Boston, MA, USA.
Benjamin P KleinstiverCenter for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA. bkleinstiver@mgh.harvard.edu.ORCID 0000-0002-5469-0655
Funding
PROJECT 4: Somatic evolution of the hematopoietic system in cardiovascular diseaseP01HL142494 · NHLBI · MASSACHUSETTS GENERAL HOSPITAL · PI Matthias Nahrendorf · 2019 to 2026
$19.5MP30 Core Grant for Vision ResearchP30EY014104 · NEI · MASSACHUSETTS EYE AND EAR INFIRMARY · PI Janey L Wiggs · 2002 to 2026
$15.1MComprehensive characterization of variants underlying heart and blood diseases with CRISPR base editingUM1HG012010 · NHGRI · MASSACHUSETTS GENERAL HOSPITAL · PI Daniel Evan Bauer, Luca Pinello · 2021 to 2026
$10.4MMaking antibody generation rapid, scalable, and democratic through machine learning and continuous evolutionR01CA260415 · NCI · UNIVERSITY OF CALIFORNIA-IRVINE · PI KRUSE, ANDREW, LIU, CHANG C · 2020 to 2024
$8.4MCRISPR/Cas9-based gene editing approaches for the treatment of USH2A-associated diseasesR01EY033107 · NEI · MASSACHUSETTS EYE AND EAR INFIRMARY · PI Qin Liu · 2022 to 2026
$3.0MMultiscale exploration of the functional non-coding genomeR35HG010717 · NHGRI · MASSACHUSETTS GENERAL HOSPITAL · PI PINELLO, LUCA · 2019 to 2023
$2.6MScalable Development of Custom Genome Editing TechnologiesDP2CA281401 · NCI · MASSACHUSETTS GENERAL HOSPITAL · PI KLEINSTIVER, BENJAMIN PETER · 2022 to 2025
$2.5MSensitive, unbiased, high-throughput, cellular GUIDE-seq-2 genome-wide activity assay for therapeutic genome editing INDsU01AI176470 · NIAID · ST. JUDE CHILDREN'S RESEARCH HOSPITAL · PI TSAI, SHENGDAR · 2023 to 2025
$1.4MNCI NIH HHS DP2 CA281401NCI NIH HHS R01 CA260415NEI NIH HHS P30 EY014104NEI NIH HHS R01 EY033107NHGRI NIH HHS R35 HG010717NHGRI NIH HHS UM1 HG012010NHLBI NIH HHS P01 HL142494NIAID NIH HHS U01 AI176470
Abstract
Engineering and characterizing proteins can be time-consuming and cumbersome, motivating the development of generalist CRISPR-Cas enzymes
Indexed as
CRISPR-Associated Protein 9CRISPR-Cas SystemsGene EditingMachine LearningNucleotide MotifsProtein EngineeringAllelesAnimalsDirected Molecular EvolutionHEK293 CellsHumansMiceNeural Networks, ComputerCRISPR-Associated Protein 9
Identifiers
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.