CRISPR & Gene-Editing Therapies
Approved and investigational CRISPR-Cas9 therapies, base and prime editing, in vivo LNP-delivered editing and bespoke editing for ultra-rare disease.
Most influential
- Selective organ targeting (SORT) nanoparticles for tissue-specific mRNA delivery and CRISPR–Cas gene editingArticle2,370 citationsdoi:10.1038/s41565-020-0669-6
- CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-ThalassemiaArticle1,960 citationsdoi:10.1056/nejmoa2031054
- Applications of genome editing technology in the targeted therapy of human diseases: mechanisms, advances and prospects
Based on engineered or bacterial nucleases, the development of genome editing technologies has opened up the possibility of directly targeting and modifying genomic sequences in almost all eukaryotic cells. Genome editing has extended our ability to elucidate the contribution of genetics to disease by promoting the creation of more accurate cellular and anim…
Abstract excerpt by Hongyi Li et al., CC-BY, https://doi.org/10.1038/s41392-019-0089-y; truncated.
Review1,720 citationsdoi:10.1038/s41392-019-0089-y - Antibiotics versus biofilm: an emerging battleground in microbial communities
Biofilm is a complex structure of microbiome having different bacterial colonies or single type of cells in a group; adhere to the surface. These cells are embedded in extracellular polymeric substances, a matrix which is generally composed of eDNA, proteins and polysaccharides, showed high resistance to antibiotics. It is one of the major causes of infectio…
Abstract excerpt by Divakar Sharma et al., CC-BY, https://doi.org/10.1186/s13756-019-0533-3; truncated.
Review1,560 citationsdoi:10.1186/s13756-019-0533-3 - Boosting CRISPR/Cas9 multiplex editing capability with the endogenous tRNA-processing systemArticle1,555 citationsdoi:10.1073/pnas.1420294112
- The next generation of CRISPR–Cas technologies and applicationsReview1,459 citationsdoi:10.1038/s41580-019-0131-5
- Enhanced prime editing systems by manipulating cellular determinants of editing outcomes
While prime editing enables precise sequence changes in DNA, cellular determinants of prime editing remain poorly understood. Using pooled CRISPRi screens, we discovered that DNA mismatch repair (MMR) impedes prime editing and promotes undesired indel byproducts. We developed PE4 and PE5 prime editing systems in which transient expression of an engineered MM…
Abstract excerpt by Chen PJ et al., CC-BY, https://doi.org/10.1016/j.cell.2021.09.018; truncated.
Article740 citationsdoi:10.1016/j.cell.2021.09.018 - Multiplex and homologous recombination–mediated genome editing in Arabidopsis and Nicotiana benthamiana using guide RNA and Cas9Article1,511 citationsdoi:10.1038/nbt.2654
- Engineered virus-like particles for efficient in vivo delivery of therapeutic proteins
Methods to deliver gene editing agents in vivo as ribonucleoproteins could offer safety advantages over nucleic acid delivery approaches. We report the development and application of engineered DNA-free virus-like particles (eVLPs) that efficiently package and deliver base editor or Cas9 ribonucleoproteins. By engineering VLPs to overcome cargo packaging, re…
Abstract excerpt by Banskota S et al., CC-BY, https://doi.org/10.1016/j.cell.2021.12.021; truncated.
Article562 citationsdoi:10.1016/j.cell.2021.12.021 - Delivering CRISPR: a review of the challenges and approaches
Gene therapy has long held promise to correct a variety of human diseases and defects. Discovery of the Clustered Regularly-Interspaced Short Palindromic Repeats (CRISPR), the mechanism of the CRISPR-based prokaryotic adaptive immune system (CRISPR-associated system, Cas), and its repurposing into a potent gene editing tool has revolutionized the field of mo…
Abstract excerpt by Christopher A. Lino et al., CC-BY, https://doi.org/10.1080/10717544.2018.1474964; truncated.
Review1,184 citationsdoi:10.1080/10717544.2018.1474964 - In vivo genome editing via CRISPR/Cas9 mediated homology-independent targeted integrationArticle1,149 citationsdoi:10.1038/nature20565
- In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophyArticle1,143 citationsdoi:10.1126/science.aad5143
- Therapeutic in vivo delivery of gene editing agents
In vivo gene editing therapies offer the potential to treat the root causes of many genetic diseases. Realizing the promise of therapeutic in vivo gene editing requires the ability to safely and efficiently deliver gene editing agents to relevant organs and tissues in vivo. Here, we review current delivery technologies that have been used to enable therapeut…
Abstract excerpt by Raguram A et al., CC-BY, https://doi.org/10.1016/j.cell.2022.03.045; truncated.
Review459 citationsdoi:10.1016/j.cell.2022.03.045 - Applications of CRISPR technologies in research and beyondArticle1,004 citationsdoi:10.1038/nbt.3659
- p53 inhibits CRISPR–Cas9 engineering in human pluripotent stem cellsArticle977 citationsdoi:10.1038/s41591-018-0050-6
Newest work
- EDIT-401 in Adults Who Require Additional Lowering of Low-Density Lipoprotein Cholesterol (LDL-C)Clinical trialNCT07894887
- Base editing-mediated exon-specific knockout of glutamine synthetase generates a high-productivity CHO cell line for antibody manufacturingArticledoi:10.1186/s40643-026-01150-0
- A Study of the Safety and Efficacy of Prime Editing (PM647a) in Participants With Alpha-1 Antitrypsin DeficiencyClinical trialNCT07887347
- Validation of a Modular Base Editing Strategy, Free-BE, in <i>Physcomitrium patens</i>, Maize, and WheatArticledoi:10.1002/pld3.70196
- cPRIME enables endogenous mapping of genetic variant effects at RNA levelArticledoi:10.1038/s41467-026-78344-9
- Individualized and Platform-Designated Gene-Editing Studies in the Registered Clinical Record: A Case-by-Case Census Against 148 Eligible StudiesArticledoi:10.7759/cureus.117975
- Mitochondrial Quality Control in Degenerative Musculoskeletal Diseases: Signaling Networks and Therapeutic LandscapesReviewdoi:10.1002/advs.78309
- Tracing model-generated DNA with position-independent watermarkingPreprintarXiv:2610.04763
- Chemically stabilized long antisense oligonucleotides perturb RNA exosome functionArticledoi:10.1093/nar/gkag990
- Validation of a Modular Base Editing Strategy, Free‐BE, in Physcomitrium patens, Maize, and WheatArticle
Clinical trials
- MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)Clinical trialNCT06128629
- HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE)Clinical trialNCT06634420
- A Long-term Follow-up Study in Participants Who Received CTX001Clinical trialNCT04208529
- A Phase 3 Study of NTLA-2001 in ATTRv-PNClinical trialNCT06672237
- Randomized Controlled Physical Activity App Intervention for Black WomenClinical trialNCT07114900
- A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell DiseaseClinical trialNCT03745287
- A Phase 2 Safety and Efficacy Study Evaluating CS-101 in Participants With β-Thalassemia MajorClinical trialNCT07489196
- Safety and Preliminary Efficacy of CTX112 in Adult Participants With Relapsed/Refractory Hematologic Autoimmune DiseaseClinical trialNCT07549698