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• Gene therapy for hypertrophic cardiomyopathy: From feasibility to a fieldwide framework.
  europepmc 2026 置信度 0.80
• Promises and potential pitfalls of in vivo CAR gene therapy.
  europepmc 2026 置信度 0.80
• A second chance for gene therapy to restore natural hearing.
  europepmc 2026 置信度 0.80
• Cross-Correction in HSC Gene Therapy for Metachromatic Leukodystrophy.
  europepmc 2026 置信度 0.80
• Is gene therapy a cure for β-thalassemia?
  europepmc 2026 置信度 0.80
• Subretinal Gene Therapy for X-Linked Retinoschisis.
  europepmc 2026 置信度 0.80
• Defining the Role of Nurses in Gene Therapy for Haemophilia.
  europepmc 2026 置信度 0.80
• A rare case of gene therapy-linked cancer.
  europepmc 2026 置信度 0.80
• Intervening in aging and related diseases with gene therapy techniques.
  europepmc 2026 置信度 0.80
• Gene Therapy for Cardiomyopathy: Tools, Targets, and Trials.
  europepmc 2026 置信度 0.80
• Gene therapy approaches for inborn errors of immunity: from bench to bedside.
  europepmc 2026 置信度 0.80
• Optimization of AAV tools to target Müller glial cells for retinal gene therapy.
  europepmc 2026 置信度 0.80
• Cardiotoxic Effects and Microdystrophin Expression after Gene Therapy for DMD.
  europepmc 2026 置信度 0.80
• Comorbid Auditory and Visual Dysfunction: From Pathogenic Genes to Gene Therapy
  europepmc 2026 置信度 0.80
• Nanoengineering Systems for Gene Therapy: Mechanisms, Modalities, and Future Directions.
  europepmc 2026 置信度 0.80
• Plugging the diastolic calcium leak: MYBPC3 fragment for CPVT gene therapy.
  europepmc 2026 置信度 0.80
• BAG3 gene therapy emerges as a potential treatment for myofibrillar myopathies.
  europepmc 2026 置信度 0.80
• Evaluating cases of possible insertional mutagenesis after AAV gene therapy.
  europepmc 2026 置信度 0.80
• The dermis as a gene therapy target: AAVrh32.33 finds an unexpected home in skin.
  europepmc 2026 置信度 0.80
• FGF21 gene therapy for healthy aging: Great promise, important questions.
  europepmc 2026 置信度 0.80
• Beyond innovation: why rare disease gene therapy needs a reset.
  europepmc 2026 置信度 0.80
• AAV vectors for cancer gene therapy.
  europepmc 2026 置信度 0.80
• Personalized gene therapy relieves severe epilepsy in two boys.
  europepmc 2026 置信度 0.80
• New Gene Therapy Strategy for β-Thalassemia.
  europepmc 2026 置信度 0.80
• Overview of Patient-Reported Outcomes in Haemophilia Gene Therapy.
  europepmc 2026 置信度 0.80
• FDA Approves Gene Therapy for Younger Children With Sickle Cell Disease.
  europepmc 2026 置信度 0.80
• The future of pediatric gene therapy: CRISPR-Cas9, AI, and personalized medicine.
  europepmc 2026 置信度 0.80
• Hematopoietic Stem-Cell Gene Therapy for Cystinosis. Reply.
  europepmc 2026 置信度 0.80
• A novel gene therapy approach for nephropathic cystinosis.
  europepmc 2026 置信度 0.80
• Gene therapy in patients with choroideremia: a meta-analysis of clinical trials.
  europepmc 2026 置信度 0.80
• Gene therapy, RNA-based drugs, and CRISPR in neuroprotection.
  europepmc 2026 置信度 0.80
• Retraction Note: Telomerase gene therapy: a remission toward cancer.
  europepmc 2026 置信度 0.80
• Activity-dependent antioxidant gene therapy protects against epileptogenesis.
  europepmc 2026 置信度 0.80
• Translational consistency of gene therapy strategies targeting inflammation in atrial fibrillation's management.
  europepmc 2026 置信度 0.80
• Gene Therapy for Heart Failure.
  europepmc 2026 置信度 0.80
• Hemophilia A gene therapy: Moving to the next chapter.
  europepmc 2026 置信度 0.80
• A gene therapy strategy for Deoxyhypusine Synthase (DHPS) syndrome
  europepmc 2026 置信度 0.80
• Parfitian Priority, Gene Therapy, and Disability.
  europepmc 2026 置信度 0.80
• Building safeguards for gene therapy in haemophilia.
  europepmc 2026 置信度 0.80
• "Just right" AAV gene therapy for spinal muscular atrophy.
  europepmc 2026 置信度 0.80
• Gene therapy for liver diseases: methods, challenges and opportunities.
  europepmc 2026 置信度 0.80
• The role of gene therapy in neural injury repair.
  europepmc 2026 置信度 0.80
• Combination S100A1 and ARC gene therapy as a treatment for DMD cardiomyopathy.
  europepmc 2026 置信度 0.80
• Exosome Nanotechnology in Molecular Medicine Advances, Applications and Challenges in Gene Therapy.
  europepmc 2026 置信度 0.80
• Rewriting the sarcomere: Gene therapy approaches for hypertrophic cardiomyopathy from bench to bedside.
  europepmc 2026 置信度 0.80
• Hemophilia in Mexico: Updated Consensus Recommendations on Diagnosis, Treatment and Gene Therapy.
  europepmc 2026 置信度 0.80
• Gaucher Disease Treated With Lentiviral-Mediated Gene Therapy: First Case.
  europepmc 2026 置信度 0.80
• Progress and challenges in intrathecal gene therapy for neurological disorders.
  europepmc 2026 置信度 0.80
• Clinical outcomes of lentiviral vector gene therapy for sickle cell disease.
  europepmc 2026 置信度 0.80
• Storage and Stability of AAV-Containing Fibrin Hydrogels for Retinal Gene Therapy.
  europepmc 2026 置信度 0.80
• Successful gene therapy for transfusion-dependent α-thalassemia: a case report.
  europepmc 2026 置信度 0.80
• Lonvoguran ziclumeran: a CRISPR-CAS9-based gene therapy for the treatment of hereditary angioedema.
  europepmc 2026 置信度 0.80
• Prescriptive Approach to Convective Perfusion of the Bilateral Thalami for Gene Therapy.
  europepmc 2026 置信度 0.80
• Gene Therapy for β-Haemoglobinopathies: From Molecular Correction to Curative Medicine.
  europepmc 2026 置信度 0.80
• Gene Therapy for Nephropathic Cystinosis.
  europepmc 2026 置信度 0.80
• Haematopoetic stem and progenitor cell gene therapy for metachromatic leukodystrophy: Mission possible!
  europepmc 2026 置信度 0.80
• [Advances in research on the molecular mechanisms and gene therapy of hereditary hearing impairment].
  europepmc 2026 置信度 0.80
• Combining modeling approaches for rapid preclinical evaluation of retinal gene therapy.
  europepmc 2026 置信度 0.80
• Restoration, Not Bypass: Otoferlin Gene Therapy and a New Era in Hearing Loss Treatment.
  europepmc 2026 置信度 0.80
• Adeno-Associated Virus Gene Therapy: Is There a Risk of Insertional Mutagenesis?
  europepmc 2026 置信度 0.80
• HSCT in SAVI: evidence for a curative hematopoietic approach and perspectives for gene therapy.
  europepmc 2026 置信度 0.80
• Promises of co-stimulatory inhibition for durable and repeatable AAV gene therapy.
  europepmc 2026 置信度 0.80
• Sickle Cell Disease: From Ancient Origins to Modern Breakthroughs in Gene Therapy.
  europepmc 2026 置信度 0.80
• Toward Safe and Effective Gene Therapy: Non-Viral Nanostructured Delivery Systems.
  europepmc 2026 置信度 0.80
• Biodegradable Acoustic Targeting for Ultrasound-Supported Gene Therapy (BATUS) in Glioblastoma.
  europepmc 2026 置信度 0.80
• Regeneron Wins FDA Approval for First Neurosensory Gene Therapy.
  europepmc 2026 置信度 0.80
• Gene Therapy for Sickle Cell Disease: Practice Recommendations from the American Society for Transplantation and Cellular Therapy and the International Society for Cell & Gene Therapy.
  europepmc 2026 置信度 0.80
• Bringing Gene Therapy Into Real World Clinical Practice.
  europepmc 2026 置信度 0.80
• Triple AAV Intein-Mediated Gene Therapy Ameliorates Dystrophic Phenotype in MDC1A Mice.
  europepmc 2026 置信度 0.80
• Gene therapy for aging: Current evidence and future directions.
  europepmc 2026 置信度 0.80
• AAV-mediated gene therapy demonstrates phenotypic rescue in a mouse model of Cockayne Syndrome.
  europepmc 2026 置信度 0.80
• Genomic landscape of inherited retinal diseases for gene therapy and clinical trials in Argentina
  europepmc 2026 置信度 0.80
• Delandistrogene moxeparvovec: gene therapy for Duchenne muscular dystrophy.
  europepmc 2026 置信度 0.80
• Perm1 Gene Therapy Mitigates PRDM16-Associated Cardiomyopathy
  europepmc 2026 置信度 0.80
• Advancing dual gene therapy for SFTPC deficiency.
  europepmc 2026 置信度 0.80
• Beyond editing: delivery systems will shape the future of gene therapy for chronic liver disease.
  europepmc 2026 置信度 0.80
• Oncology Nurses' Role in Cell and Gene Therapy: Future Expansions and Evolving Foundations.
  europepmc 2026 置信度 0.80
• Successful live birth in a woman with partial 17α-hydroxylase/17,20 lyase deficiency: a case report.
  17a-hydroxylase/17,20-lyase deficiency (17-OHD) is a rare form of congenital adrenal hyperplasia caused by mutations in the CYP17A1 gene, resulting in impaired activity of the corresponding enzymes. Its typical manifestations include hypertension, hypokalemia,…
  pubmed Wu L, Wang P, Li J, Yu X 等 2026 置信度 0.82
• Case Report: Overlap of long QT syndrome and catecholaminergic polymorphic ventricular tachycardia in two Chinese children with CALM2-related calmodulinopathy.
  Calmodulinopathy is a rare CALM -related hereditary channelopathy presenting with long QT syndrome (LQTS), less commonly with catecholaminergic polymorphic ventricular tachycardia (CPVT) or LQTS/CPVT overlap. It carries high mortality and limited data from Chi…
  pubmed Wu J, Qi J, Liao Y 2026 置信度 0.82
• Efficient and precise programmable DNA knock-in without double-strand breaks.
  Programmable gene knock-in holds substantial promise for treating genetic diseases and advancing cell therapies. However, achieving precise and efficient kilobase-scale DNA fragment integration remains challenging 1,2 . Here we report CRISPR kilobase-scale nic…
  pubmed Gao Y, Ma Y, Yu K, Liu Y 等 2026 Jul 22 置信度 0.82
• AAV-CRISPR Gene Editing Is Negated by Pre-existing Immunity to Cas9.
  europepmc 2020 置信度 0.80
• Sensitization of The Antibiotic Resistant M. smegmatis Bacteria Using CRISPR/FnCpf1 Gene Editing
  crossref Hasret Araz 2023-12-16T09:55:21Z 置信度 0.70
• CRISPR and Gene Editing: Ethical and Scientific Perspectives
  CRISPR-Cas9 has emerged as one of the most transformative tools in genetic engineering, enabling scientists to perform precise, efficient, and cost-effective gene modifications. This revolutionary gene-editing technology has broad applications in medicine, agr…
  crossref Jennifer Doudna 2025-04-19T05:40:58Z 置信度 0.70
• CRISPR/Cas: A Genome-Editing Tool for Crops Improvement
  crossref Nishtha Goel, Asha Arora, Ashwani Kumar 2024-03-18T08:02:35Z 置信度 0.70
• Targeted pinpoint gene editing tool, CRISPR/Cas9: A Review
  CRISPR has emanated as a powerful tool for targeted, precision genome editing and is extensively captivating biomedical research world nowadays. Being more precise, faster and cheaper than predecessor DNA editing strategies like ZFN (Zinc Finger Nucleases) and…
  crossref Hafsa Tahir, Rashid Saif, Talha Tamseel, Fraz Ahmad 2026-01-19T10:22:53Z 置信度 0.70
• Electroporation-Based CRISPR/Cas9 Gene Editing Using Cas9 Protein and Chemically Modified sgRNAs
  crossref Anders Laustsen, Rasmus O. Bak 2019-03-25T09:03:05Z 置信度 0.70
• Non-viral Alternatives to Delivery of CRISPR-Cas Gene Editing Components
  crossref Kate Senger, Benjamin Haley 2021-12-15T16:42:46Z 置信度 0.70
• CRISPR-Cas Systems in Agriculture
  crossref Farkhanda Jabeen, Noreen Aasim, Farheen Shafique 2025-07-04T10:41:22Z 置信度 0.70
• Selectively cleavable CRISPR guide RNAs for safe therapeutic gene editing
  crossref Minghong Zhong 2025-09-08T16:44:04Z 置信度 0.70
• Gene Hackers
  crossref 2016-10-25T11:35:06Z 置信度 0.70
• CRISPR Gene Editing Meets the Art World.
  europepmc Miguel Ángel Medina 2018 置信度 0.80
• The Gene Trade
  crossref 2018-04-19T06:01:55Z 置信度 0.70
• Improvement of Vegetable Crops Using CRISPR-Cas9 as A Gene Editing Tool
  crossref Dilawar Khan, Kumail Ali Rizvi, Asim Gulzar, Tayyaba Zainab 2026-04-01T14:14:25Z 置信度 0.70
• Advancement in CRISPR/Cas9 Genome Editing in Field of Crop Improvement
  crossref Husnain Rauf 2026-04-01T14:14:25Z 置信度 0.70
• Efficient Gene Editing of Human Induced Pluripotent Stem Cells Using CRISPR/Cas9
  crossref Saniye Yumlu, Sanum Bashir, Jürgen Stumm, Ralf Kühn 2019-03-25T09:03:05Z 置信度 0.70
• CRISPR/Cas9 Gene Editing in Hepatocellular Carcinoma: Current Progress and Future Perspectives
  crossref Hanyu Zheng 2026-01-27T11:57:46Z 置信度 0.70
• Surveying the State of CRISPR and Gene Editing
  crossref Rodolphe Barrangou 2024-06-26T10:00:51Z 置信度 0.70
• Transient CRISPR-Cas delivery for therapeutic gene editing in the retina
  Transfert transitoire de CRISPR‐Cas pour l'édition de gène dans la rétine L'édition du gène par CRISPR-Cas s'est révélée prometteuse pour le traitement des dystrophies rétiniennes (DR). L'administration transitoire de la protéine Cas9 et son ARNg sous forme de…
  crossref Catherine Botto 2026-04-07T07:44:06Z 置信度 0.70
• Figure 1: Workflow for the use of CRISPR-Cas9-based gene editing in plants.
  crossref 2024-06-07T03:19:18Z 置信度 0.70
• CRISPR controversy raises questions about gene-editing technique
  crossref Ian Haydon 2025-08-26T06:40:34Z 置信度 0.70