# CRISPR & Gene-Editing Therapies Domain: Advanced Medicine · 264 papers indexed · newest 2026-10-26 Approved and investigational CRISPR-Cas9 therapies, base and prime editing, in vivo LNP-delivered editing and bespoke editing for ultra-rare disease. Key concepts: CRISPR, base editing, prime editing ## Most influential - **[Selective organ targeting (SORT) nanoparticles for tissue-specific mRNA delivery and CRISPR–Cas gene editing](https://space-kitty.com/papers/doi:10.1038/s41565-020-0669-6)**. Qiang Cheng, Tuo Wei, Lukas Farbiak et al. · 2020 · Nature Nanotechnology (doi:10.1038/s41565-020-0669-6 · 2,370 citations). Source: - **[CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia](https://space-kitty.com/papers/doi:10.1056/nejmoa2031054)**. Haydar Frangoul, David Altshuler, Maria Domenica Cappellini et al. · 2020 · New England Journal of Medicine (doi:10.1056/nejmoa2031054 · 1,960 citations). Source: - **[Applications of genome editing technology in the targeted therapy of human diseases: mechanisms, advances and prospects](https://space-kitty.com/papers/doi:10.1038/s41392-019-0089-y)**. Hongyi Li, Yang Yang, Weiqi Hong et al. · 2020 · Signal Transduction and Targeted Therapy (doi:10.1038/s41392-019-0089-y · 1,720 citations). Source: > 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 animal models of pathological processes and has begun to show extraordinary potential in a variety of fields, ranging from basic research to applied biotechnology and biomedical research. Recent progress in developing programmable nucleases, su… > _Abstract excerpt by Hongyi Li et al., CC-BY, https://doi.org/10.1038/s41392-019-0089-y; truncated._ - **[Antibiotics versus biofilm: an emerging battleground in microbial communities](https://space-kitty.com/papers/doi:10.1186/s13756-019-0533-3)**. Divakar Sharma, Lama Misba, Asad Ullah Khan · 2019 · Antimicrobial Resistance and Infection Control (doi:10.1186/s13756-019-0533-3 · 1,560 citations). Source: > 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 infection persistence especially in nosocomial settings through indwelling devices. Quorum sensing plays an important role in regulating the biofilm formation. There are many approaches being used to control infections by suppressing its formation … > _Abstract excerpt by Divakar Sharma et al., CC-BY, https://doi.org/10.1186/s13756-019-0533-3; truncated._ - **[Boosting CRISPR/Cas9 multiplex editing capability with the endogenous tRNA-processing system](https://space-kitty.com/papers/doi:10.1073/pnas.1420294112)**. Kabin Xie, Bastian Minkenberg, Yinong Yang · 2015 · Proceedings of the National Academy of Sciences (doi:10.1073/pnas.1420294112 · 1,555 citations). Source: - **[The next generation of CRISPR–Cas technologies and applications](https://space-kitty.com/papers/doi:10.1038/s41580-019-0131-5)**. Adrian Pickar‐Oliver, Charles A. Gersbach · 2019 · Nature Reviews Molecular Cell Biology (doi:10.1038/s41580-019-0131-5 · 1,459 citations). Source: - **[Enhanced prime editing systems by manipulating cellular determinants of editing outcomes](https://space-kitty.com/papers/doi:10.1016/j.cell.2021.09.018)**. Chen PJ, Hussmann JA, Yan J et al. · 2021 · Cell (doi:10.1016/j.cell.2021.09.018 · 740 citations). Source: > 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 MMR-inhibiting protein enhances the efficiency of substitution, small insertion, and small deletion prime edits by an average 7.7-fold and 2.0-fold compared to PE2 and PE3 systems, respectively, while improving edit/indel ratios by 3.4-fold i… > _Abstract excerpt by Chen PJ et al., CC-BY, https://doi.org/10.1016/j.cell.2021.09.018; truncated._ - **[Multiplex and homologous recombination–mediated genome editing in Arabidopsis and Nicotiana benthamiana using guide RNA and Cas9](https://space-kitty.com/papers/doi:10.1038/nbt.2654)**. Jianfeng Li, Julie Erin Norville, John Aach et al. · 2013 · Nature Biotechnology (doi:10.1038/nbt.2654 · 1,511 citations). Source: - **[Engineered virus-like particles for efficient in vivo delivery of therapeutic proteins](https://space-kitty.com/papers/doi:10.1016/j.cell.2021.12.021)**. Banskota S, Raguram A, Suh S et al. · 2022 · Cell (doi:10.1016/j.cell.2021.12.021 · 562 citations). Source: > 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, release, and localization bottlenecks, we developed fourth-generation eVLPs that mediate efficient base editing in several primary mouse and human cell types. Using different glycoproteins in eVLPs alters their cellular tropism. Single inject… > _Abstract excerpt by Banskota S et al., CC-BY, https://doi.org/10.1016/j.cell.2021.12.021; truncated._ - **[Delivering CRISPR: a review of the challenges and approaches](https://space-kitty.com/papers/doi:10.1080/10717544.2018.1474964)**. Christopher A. Lino, Jason C. Harper, James P. Carney et al. · 2018 · Drug Delivery (doi:10.1080/10717544.2018.1474964 · 1,184 citations). Source: > 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 molecular biology and generated excitement for new and improved gene therapies. Additionally, the simplicity and flexibility of the CRISPR/Cas9 site-specific nuclease system has led to its widespread use in many biological research areas incl… > _Abstract excerpt by Christopher A. Lino et al., CC-BY, https://doi.org/10.1080/10717544.2018.1474964; truncated._ - **[In vivo genome editing via CRISPR/Cas9 mediated homology-independent targeted integration](https://space-kitty.com/papers/doi:10.1038/nature20565)**. Keiichiro Suzuki, Yuji Tsunekawa, Reyna Hernández‐Benítez et al. · 2016 · Nature (doi:10.1038/nature20565 · 1,149 citations). Source: - **[In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy](https://space-kitty.com/papers/doi:10.1126/science.aad5143)**. Christopher E. Nelson, Chady H. Hakim, David G. Ousterout et al. · 2015 · Science (doi:10.1126/science.aad5143 · 1,143 citations). Source: - **[Therapeutic in vivo delivery of gene editing agents](https://space-kitty.com/papers/doi:10.1016/j.cell.2022.03.045)**. Raguram A, Banskota S, Liu DR · 2022 · Cell (doi:10.1016/j.cell.2022.03.045 · 459 citations). Source: > 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 therapeutic in vivo gene editing, including viral vectors, lipid nanoparticles, and virus-like particles. Since no single delivery modality is likely to be appropriate for every possible application, we compare the benefits and drawbacks of each met… > _Abstract excerpt by Raguram A et al., CC-BY, https://doi.org/10.1016/j.cell.2022.03.045; truncated._ - **[Applications of CRISPR technologies in research and beyond](https://space-kitty.com/papers/doi:10.1038/nbt.3659)**. Rodolphe Barrangou, Jennifer A. Doudna · 2016 · Nature Biotechnology (doi:10.1038/nbt.3659 · 1,004 citations). Source: - **[p53 inhibits CRISPR–Cas9 engineering in human pluripotent stem cells](https://space-kitty.com/papers/doi:10.1038/s41591-018-0050-6)**. Robert J. Ihry, Kathleen A. Worringer, Max R. Salick et al. · 2018 · Nature Medicine (doi:10.1038/s41591-018-0050-6 · 977 citations). Source: ## Newest - **[EDIT-401 in Adults Who Require Additional Lowering of Low-Density Lipoprotein Cholesterol (LDL-C)](https://space-kitty.com/papers/nct:NCT07894887)**. Editas Medicine, Inc. · 2026 · ClinicalTrials.gov (NCT07894887). Source: - **[Base editing-mediated exon-specific knockout of glutamine synthetase generates a high-productivity CHO cell line for antibody manufacturing](https://space-kitty.com/papers/doi:10.1186/s40643-026-01150-0)**. Pu T, Wang C, Lou H et al. · 2026 · Bioresources and bioprocessing (doi:10.1186/s40643-026-01150-0). Source: - **[A Study of the Safety and Efficacy of Prime Editing (PM647a) in Participants With Alpha-1 Antitrypsin Deficiency](https://space-kitty.com/papers/nct:NCT07887347)**. Prime Medicine, Inc. · 2026 · ClinicalTrials.gov (NCT07887347). Source: - **[Validation of a Modular Base Editing Strategy, Free-BE, in \Physcomitrium patens\, Maize, and Wheat](https://space-kitty.com/papers/doi:10.1002/pld3.70196)**. Guyon-Debast A, Javelle M, Perroud PF et al. · 2026 · Plant direct (doi:10.1002/pld3.70196). Source: - **[cPRIME enables endogenous mapping of genetic variant effects at RNA level](https://space-kitty.com/papers/doi:10.1038/s41467-026-78344-9)**. El Kassem G, Westphal C, Doebel M et al. · 2026 · Nature communications (doi:10.1038/s41467-026-78344-9). Source: - **[Individualized and Platform-Designated Gene-Editing Studies in the Registered Clinical Record: A Case-by-Case Census Against 148 Eligible Studies](https://space-kitty.com/papers/doi:10.7759/cureus.117975)**. Li OM, Li Y · 2026 · Cureus (doi:10.7759/cureus.117975). Source: - **[Mitochondrial Quality Control in Degenerative Musculoskeletal Diseases: Signaling Networks and Therapeutic Landscapes](https://space-kitty.com/papers/doi:10.1002/advs.78309)**. Gao H, Cheng Z, Chen X et al. · 2026 · Advanced science (Weinheim, Baden-Wurttemberg, Germany) (doi:10.1002/advs.78309). Source: - **[Tracing model-generated DNA with position-independent watermarking](https://space-kitty.com/papers/arxiv:2610.04763)**. Kimon Antonios Provatas1, Christos Galanopoulos, Ilias Georgakopoulos-Soares · 2026 (arXiv:2610.04763). Source: - **[Chemically stabilized long antisense oligonucleotides perturb RNA exosome function](https://space-kitty.com/papers/doi:10.1093/nar/gkag990)**. Hofacker DT, Gackstatter S, Deisling V et al. · 2026 · Nucleic acids research (doi:10.1093/nar/gkag990). Source: - **[Validation of a Modular Base Editing Strategy, Free‐BE, in Physcomitrium patens, Maize, and Wheat](https://space-kitty.com/papers/pmc:PMC13670207)**. Guyon‐Debast A, Javelle M, Perroud P et al. · 2026 · Plant direct. Source: ## Clinical trials - **[MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)](https://space-kitty.com/papers/nct:NCT06128629)**. Intellia Therapeutics · 2023 · ClinicalTrials.gov (NCT06128629). Source: - **[HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE)](https://space-kitty.com/papers/nct:NCT06634420)**. Intellia Therapeutics · 2024 · ClinicalTrials.gov (NCT06634420). Source: - **[A Long-term Follow-up Study in Participants Who Received CTX001](https://space-kitty.com/papers/nct:NCT04208529)**. Vertex Pharmaceuticals Incorporated · 2019 · ClinicalTrials.gov (NCT04208529). Source: - **[A Phase 3 Study of NTLA-2001 in ATTRv-PN](https://space-kitty.com/papers/nct:NCT06672237)**. Intellia Therapeutics · 2024 · ClinicalTrials.gov (NCT06672237). Source: - **[Randomized Controlled Physical Activity App Intervention for Black Women](https://space-kitty.com/papers/nct:NCT07114900)**. Stony Brook University · 2025 · ClinicalTrials.gov (NCT07114900). Source: - **[A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease](https://space-kitty.com/papers/nct:NCT03745287)**. Vertex Pharmaceuticals Incorporated · 2018 · ClinicalTrials.gov (NCT03745287). Source: - **[A Phase 2 Safety and Efficacy Study Evaluating CS-101 in Participants With β-Thalassemia Major](https://space-kitty.com/papers/nct:NCT07489196)**. CorrectSequence Therapeutics Co., Ltd · 2026 · ClinicalTrials.gov (NCT07489196). Source: - **[Safety and Preliminary Efficacy of CTX112 in Adult Participants With Relapsed/Refractory Hematologic Autoimmune Disease](https://space-kitty.com/papers/nct:NCT07549698)**. CRISPR Therapeutics AG · 2026 · ClinicalTrials.gov (NCT07549698). Source: --- Space Kitty Archive · https://space-kitty.com · API: https://space-kitty.com/api/v1 · MCP: https://space-kitty.com/mcp · Premium: $0.20/request via x402 (Base Sepolia (testnet)). Metadata: arXiv (CC0), OpenAlex (CC0), Europe PMC (CC BY/CC0 works), ClinicalTrials.gov.