Try our beta test site
IMPORTANT: Listing of a study on this site does not reflect endorsement by the National Institutes of Health. Talk with a trusted healthcare professional before volunteering for a study. Read more...
179 studies found for:    "gene therapy" OR "gene transfer" OR "virus delivery" | Open Studies
Show Display Options
Rank Status Study
1 Recruiting Gene Transfer for Patients With Sickle Cell Disease
Conditions: Sickle Cell Disease;   Sickle Cell Anemia
Intervention: Genetic: Gene transfer
2 Recruiting Long-Term Follow-Up of Recipient of Gene Transfer Research
Conditions: Retroviridae Infections;   Cancer
Interventions: Other: Blood Tests;   Behavioral: Questionnaires
3 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
4 Recruiting Gene Therapy for X-CGD
Condition: X-linked Chronic Granulomatous Disease
Intervention: Genetic: ex-vivo gene-therapy
5 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
6 Recruiting A Gene Therapy Study for Homozygous Familial Hypercholesterolemia (HoFH)
Condition: Homozygous Familial Hypercholesterolemia
Intervention: Biological: AAV directed hLDLR gene therapy
7 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
8 Recruiting Study of RS1 Ocular Gene Transfer for X-linked Retinoschisis
Condition: Gene Transfer
Intervention: Biological: RS1 AAV Vector
9 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
10 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
11 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
12 Recruiting Batten CLN6 Gene Therapy
Conditions: Batten Disease;   CLN6
Intervention: Drug: scAVV9.CB.CLN6
13 Recruiting RGX-314 Gene Therapy for Neovascular AMD Trial
Conditions: Neovascular Age-related Macular Degeneration;   Wet Age-related Macular Degeneration
Intervention: Biological: RGX-314
14 Not yet recruiting Safety Study of Gene Transfer Vector to Treat Alpha1-antitrypsin (A1AT) Deficiency
Condition: Alpha 1-Antitrypsin Deficiency
Intervention: Genetic: ADVM-043
15 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
16 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
17 Recruiting Gene Therapy for WAS
Condition: Wiskott-Aldrich Syndrome
Intervention: Genetic: Autologous CD34 positive cells transduced with a lentiviral vector containing human WAS gene
18 Not yet recruiting Gene Therapy for Haemophilia A.
Condition: Hemophilia A
Intervention: Biological: AAV2/8-HLP-FVIII-V3
19 Recruiting Gene Therapy for WAS Follow-up
Condition: Wiskott-Aldrich Syndrome
Intervention: Genetic: Autologous CD34+ cells transduced with WASP lentiviral vector
20 Recruiting Gene Therapy for Wiskott-Aldrich Syndrome (WAS)
Condition: Wiskott-Aldrich Syndrome
Intervention: Genetic: Autologous CD34 positive cells transduced with a lentiviral vector containing human WAS gene

   Previous Page Studies Shown (1-20) Next Page (21-40) Show next page of results    Last Page
Study has passed its completion date and status has not been verified in more than two years.