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125 studies found for:    Open Studies | "gene therapy"
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Show studies that were first received in the last 14 daysShow studies that were first received in the last 14 days
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Rank Status Study
1 Recruiting Gene Therapy for X-CGD
Condition: X-linked Chronic Granulomatous Disease
Intervention: Genetic: ex-vivo gene-therapy
2 Not yet recruiting p53 Gene Therapy in Treatment of Diabetes Concurrent With Hepatocellular Carcinoma
Conditions: HCC;   Diabetes
Interventions: Drug: p53 gene therapy;   Drug: Trans-catheter embolization
3 Recruiting A Gene Therapy Study for Homozygous Familial Hypercholesterolemia (HoFH)
Condition: Homozygous Familial Hypercholesterolemia
Intervention: Biological: AAV directed hLDLR gene therapy
4 Recruiting Effect of Intratumoral Injection of Gene Therapy for Locally Advanced Pancreatic Cancer
Condition: Pancreatic Adenocarcinoma
Interventions: Drug: Gene Therapy product CYL-02;   Drug: Gemcitabine
5 Recruiting Gene Transfer for Patients With Sickle Cell Disease
Conditions: Sickle Cell Disease;   Sickle Cell Anemia
Intervention: Genetic: Gene transfer
6 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
7 Recruiting Gene Therapy for X-linked Chronic Granulomatous Disease
Condition: X-Linked Chronic Granulomatous Disease
Intervention: Genetic: X vivo gene therapy
8 Recruiting Gene Therapy for X-linked Chronic Granulomatous Disease (X-CGD)
Condition: X-Linked Chronic Granulomatous Disease
Intervention: Genetic: X vivo gene therapy
9 Recruiting Gene Transfer for Severe Combined Immunodeficiency, X-linked (SCID-X1) Using a Self-inactivating (SIN) Gammaretroviral Vector
Condition: Severe Combined Immunodeficiency
Intervention: Biological: Gene transfer
10 Recruiting Long-Term Follow-Up of Recipient of Gene Transfer Research
Conditions: Retroviridae Infections;   Cancer
Interventions: Other: Blood Tests;   Behavioral: Questionnaires
11 Recruiting RGX-314 Gene Therapy for Neovascular AMD Trial
Conditions: Neovascular Age-related Macular Degeneration;   Wet Age-related Macular Degeneration
Intervention: Biological: RGX-314
12 Recruiting Dose-Escalation Study Of A Self Complementary Adeno-Associated Viral Vector For Gene Transfer in Hemophilia B
Condition: Hemophilia B
Interventions: Genetic: Gene Transfer;   Drug: scAAV2/8-LP1-hFIXco
13 Recruiting Long Term Follow Up Of Patients Who Have Received Gene Therapy Or Gene Marked Products
Conditions: Severe Combined Immunodeficiency;   Malignancy, Hematologic;   Neuroblastoma;   Neoplasm;   Mucopolysaccharidosis I
Intervention: Procedure: Venipuncture
14 Recruiting A Gene Therapy Study for Hemophilia B
Condition: Hemophilia B
Intervention: Genetic: SPK-9001
15 Recruiting Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID)
Conditions: X-linked Severe Combined Immunodeficiency;   XSCID;   SCID-X1;   Gamma C-Deficient SCID
Interventions: Other: Gene-modified CD34+ Hematopoietic stem cells;   Drug: Busulfan
16 Recruiting Safety and Efficacy Study of Gene Therapy for The Treatment of Leber's Hereditary Optic Neuropathy
Condition: Leber Hereditary Optic Neuropathy
Interventions: Drug: rAAV2-ND4;   Drug: normal saline
17 Recruiting Gene Therapy for WAS Follow-up
Condition: Wiskott-Aldrich Syndrome
Intervention: Genetic: Autologous CD34+ cells transduced with WASP lentiviral vector
18 Not yet recruiting Safety and Efficacy Study of Neovasculgen (Pl-VEGF165) Gene Therapy in Patients With Peripheral Nerve Injury
Condition: Peripheral Nerve Injury
Intervention: Drug: Neovasculgen
19 Recruiting Gene Therapy for Netherton Syndrome
Condition: Netherton Syndrome
Intervention: Genetic: One 20cm2/10cm2 autologous skin sheet graft
20 Recruiting Gene Therapy After Frontline Chemotherapy in Treating Patients With AIDS-Related Non-Hodgkin Lymphoma
Conditions: AIDS-related Non-Hodgkin Lymphoma;   AIDS-related Plasmablastic Lymphoma;   AIDS-related Primary Effusion Lymphoma;   HIV Infection
Interventions: Drug: busulfan;   Biological: lentivirus vector rHIV7-shI-TAR-CCR5RZ-transduced hematopoietic progenitor cells;   Other: pharmacological study;   Other: laboratory biomarker analysis

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