gene-therapy

Documents about gene-therapy

• Reprogramming human T cell function and specificity with non-viral genome targeting#814708eScholarship
• Phase II Clinical Study of JS207 (PD-1/VEGF Bispecific Antibody) Combination Therapies in Patients With Driver Gene-Positive, Advanced Non-Small Cell Lung Cancer After Failure of TKI Therapy#993000ClinicalTrials.gov
• Common alleles at 6q25.1 and 1p11.2 are associated with breast cancer risk for BRCA1 and BRCA2 mutation carriers#179022Lund University Publications
• CALM-AF-AI: Counteracting Age-related Loss of Muscle With AAV-Follistatin Combined With Angiogenesis-Inducing VEGF Plasmid Gene Therapy#807185ClinicalTrials.gov
• NG101 AAV Gene Therapy in Subjects With Wet Age-Related Macular Degeneration#845848ClinicalTrials.gov
• Stem Cell (Modified Bone Marrow) Transplantation in HIV-Infected Patients With Blood Cancer#908456ClinicalTrials.gov
• BIN1 overexpression rescues cardiac but not skeletal muscle defects in a mouse model of caveolinopathy#971189HAL (France)
• First-in-Human Clinical Trial of STUP-001, an In Vivo Direct Cell Conversion Gene Therapy for AIS-A/B Chronic Spinal Cord Injury#881474ClinicalTrials.gov
• Efficacy and Safety Study of Bilateral IVT Injection of GS010 at Two Dose Levels in LHON Patients#966529ClinicalTrials.gov
• Lenalidomide and Vaccine Therapy in Treating Patients With Early-Stage Asymptomatic Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma#900011ClinicalTrials.gov
• An IIT Clinical Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of a Single Intrathecal Injection of SNUG01 in Patients with Amyotrophic Lateral Sclerosis#970063ClinicalTrials.gov
• Clinical Study on the Prevention of Driver Gene Negative Ib-IIIb Lung Cancer Recurrence and Metastasis by Staged Chinese Herbal Medicine Combined With Chemotherapy and Immune Checkpoint Inhibitors#883751ClinicalTrials.gov
• Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg#913184ClinicalTrials.gov
• CD19.CAR Allogeneic NKT for Patients With Relapsed or Refractory B-Cell Malignancies (ANCHOR)#861685ClinicalTrials.gov
• Generation of a New Immunodeficient Rat Model of Retinal Degeneration With LSL TdTomato Reporter and TdTomato-Pcp2 Expression#814581eScholarship
• A Phase 1/2, First-in-Human Dose Escalation/Expansion Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of a Subretinal Injection of SB-007 in Subjects With Stargardt Disease (STGD1)#828135ClinicalTrials.gov
• Second-Line Sacituzumab Tirumotecan Plus Anlotinib for Advanced Driver Gene-Negative Non-Squamous NSCLC#979982ClinicalTrials.gov
• Figure 1 from: Abuhaliema A, Gharaibeh L, Jaradat E, Hammad M, Alshaer W (2026) Non-coding RNAs in gene therapy: mechanisms, delivery strategies, and clinical translation. Pharmacia 73: 1-25. https://doi.org/10.3897/pharmacia.73.e173259#210307Zenodo (CERN)
• Gene Therapy in Treating Patients With Cancer#901580ClinicalTrials.gov
• Gene Therapy for CD19-Positive Hematologic Malignancies (SENTRY-CD19)#904578ClinicalTrials.gov
• Intranasal AAV9-PHP.eB Gene Therapy in Cerebral Palsy (CP) & Hypoxic Ischemic Encephalopathy (HIE)#832442ClinicalTrials.gov
• Sorafenib Tosylate and Chemotherapy in Treating Older Patients With Acute Myeloid Leukemia#979436ClinicalTrials.gov
• Treating Early-stage Non-Small Cell Lung Cancer With Durvalumab and Radiation Therapy#893133ClinicalTrials.gov
• Gene Therapy and Chemotherapy in Treating Patients With Advanced Solid Tumors or Non-Hodgkin's Lymphoma#841974ClinicalTrials.gov
• Gene Therapy in Treating Patients With Unresectable, Recurrent, or Refractory Head and Neck Cancer#913690ClinicalTrials.gov