31 studies found for:    "gene therapy" OR "gene transfer" OR "virus delivery" | Open Studies | Studies updated in the last 30 days
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Rank Status Study
1 Recruiting Study of RS1 Ocular Gene Transfer for X-linked Retinoschisis
Condition: Gene Transfer
Intervention: Biological: RS1 AAV Vector
2 Recruiting Study of Gene Therapy Using a Lentiviral Vector to Treat X-linked Chronic Granulomatous Disease
Condition: Granulomatous Disease, Chronic, X-linked
Intervention: Biological: Lentiviral G1XCGD Gene Therapy
3 Recruiting Screening Protocol for a Gene Therapy Trial in Subjects With Homozygous Familial Hypercholesterolemia
Condition: Hypercholesterolemia, Familial
Intervention:
4 Recruiting Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants
Condition: Severe Combined Immunodeficiency Disease, X-linked
Interventions: Genetic: CL20-4i-EF/a-hyc-OPT;   Drug: Busulfan
5 Not yet recruiting Gene Therapy for Haemophilia A.
Condition: Hemophilia A
Intervention: Biological: AAV2/8-HLP-FVIII-V3
6 Recruiting WT1 TCR Gene Therapy for Leukaemia: A Phase I/II Safety and Toxicity Study
Conditions: Acute Myeloid Leukaemia;   Chronic Myeloid Leukaemia
Intervention: Genetic: WT1 TCR-transduced T cells
7 Recruiting A Gene Transfer Study for Hemophilia A
Condition: Hemophilia A
Intervention: Genetic: SPK-8011
8 Recruiting A Gene Therapy Study for Hemophilia B
Condition: Hemophilia B
Intervention: Genetic: SPK-9001
9 Not yet recruiting Trial of Stereotactic Body Radiation and Gene Therapy Before Nivolumab for Metastatic Non-Small Cell Lung Carcinoma
Conditions: Lung Squamous Cell Carcinoma Stage IV;   Nonsquamous Nonsmall Cell Neoplasm of Lung
Interventions: Biological: ADV/HSV-tk;   Drug: Valacyclovir;   Radiation: SBRT;   Drug: nivolumab
10 Recruiting Safety Study of an Adeno-associated Virus Vector for Gene Therapy of Leber's Hereditary Optic Neuropathy
Condition: Leber's Hereditary Optic Neuropathy
Interventions: Drug: injection of scAAV2-P1ND4v2 5.00x10e9 vg (Low),;   Drug: injection of scAAV2-P1ND4v2 2.46X10e10 vg (Med);   Drug: injection of scAAV2-P1ND4v2 1.0X10e11vg (High)
11 Recruiting Intrathecal Administration of scAAV9/JeT-GAN for the Treatment of Giant Axonal Neuropathy
Conditions: Giant Axonal Neuropathy;   Gene Transfer
Intervention: Drug: Intrathecal Delivery of scAAV9/JeT-GAN
12 Recruiting Phase I/II Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA
Condition: Mucopolysaccharidosis Type 3 A Sanfilippo Syndrome
Intervention: Genetic: scAAV9.U1a.hSGSH
13 Recruiting Study With T-cel Receptor Gene Therapy in Metastatic Melanoma
Conditions: Stage IV Skin Melanoma;   Eye; Melanoma
Interventions: Biological: TCR transduced T-cells;   Procedure: Biopsy;   Procedure: Blood taking
14 Recruiting AAVRh.10 Administered to Children With Late Infantile Neuronal Ceroid Lipofuscinosis
Conditions: Late Infantile Neuronal Ceroid Lipofuscinosis;   Batten Disease
Intervention: Biological: AAVrh.10CUCLN2
15 Recruiting Long-Term Safety, Tolerability, and Efficacy of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B
Condition: Hemophilia B
Intervention:
16 Recruiting Stem Cell Gene Therapy for Sickle Cell Disease
Condition: Sickle Cell Disease
Intervention: Genetic: βAS3-FB vector transduced BM CD34+ cells
17 Recruiting Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset OTC Deficiency
Condition: Ornithine Transcarbamylase (OTC) Deficiency
Intervention: Genetic: scAAV8OTC
18 Recruiting Study of Combined SGT-53 Plus Gemcitabine/Nab-Paclitaxel for Metastatic Pancreatic Cancer
Condition: Metastatic Pancreatic Cancer
Interventions: Genetic: SGT-53;   Drug: nab-paclitaxel;   Drug: Gemcitabine
19 Not yet recruiting Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I
Condition: MPS I
Intervention: Genetic: SB-318
20 Recruiting Pilot and Feasibility Trial of Plerixafor for Hematopoietic Stem Cell (HSC) Mobilization in Patients With Sickle Cell Disease Pilot and Feasibility Trial of Plerixafor for Hematopoietic Stem Cell (HSC) Mobilization in Patients With Sickle Cell Disease
Condition: Sickle Cell Disease Without Crisis
Intervention: Drug: Plerixafor

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Study has passed its completion date and status has not been verified in more than two years.