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74 studies found for:    "gene therapy" OR "gene transfer" OR "virus delivery" | adeno associated
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Rank Status Study
1 Terminated Safety of a New Type of Treatment Called Gene Transfer for the Treatment of Severe Hemophilia B
Condition: Hemophilia B
Intervention: Genetic: Adeno-Associated Viral with Human Factor IX
2 Active, not recruiting Open-Label Single Ascending Dose of Adeno-associated Virus Serotype 8 Factor IX Gene Therapy in Adults With Hemophilia B
Condition: Hemophilia B
Intervention: Biological: AskBio009
3 Recruiting A Gene Therapy Study for Hemophilia B
Condition: Hemophilia B
Intervention: Genetic: SPK-9001
4 Active, not recruiting Gene Therapy for Blindness Caused by Choroideremia
Condition: Choroideremia
Intervention: Drug: rAAV2.REP1
5 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
6 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)
7 Completed Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease
Condition: Pompe Disease
Interventions: Drug: rAAV1-CMV-GAA (study agent) Administration;   Other: RMST
8 Recruiting A Gene Transfer Study for Hemophilia A
Condition: Hemophilia A
Intervention: Genetic: SPK-8011
9 Active, not recruiting Safety Study of a Gene Transfer Vector for Children With Late Infantile Neuronal Ceroid Lipofuscinosis
Conditions: Batten Disease;   Late Infantile Neuronal Ceroid Lipofuscinosis
Intervention: Genetic: AAV2CUhCLN2
10 Completed CERE-110 in Subjects With Mild to Moderate Alzheimer's Disease
Condition: Alzheimer's Disease
Intervention: Genetic: CERE-110: Adeno-Associated Virus Delivery of NGF
11 Recruiting Safety of a Single Administration of AAV2hAQP1, an Adeno-Associated Viral Vector Encoding Human Aquaporin-1 to One Parotid Salivary Gland in People With Irradiation-Induced Parotid Salivary Hypofunction
Conditions: Squamous Cell Head and Neck Cancer;   Radiation Induced Xerostomia;   Salivary Hypofunction
Intervention: Biological: AAV2hAQP1
12 Recruiting Intracerebral Gene Therapy for Children With Early Onset Forms of Metachromatic Leukodystrophy
Condition: Metachromatic Leukodystrophy
Intervention: Genetic: intracerebral administration of AAVrh.10cuARSA
13 Unknown  Phase I Randomized Study of Adeno-Associated Virus-CFTR Vector in Patients With Cystic Fibrosis
Condition: Cystic Fibrosis
Intervention: Drug: Adeno-associated virus-CFTR vector
14 Recruiting Trial of Ocular Subretinal Injection of a Recombinant Adeno-Associated Virus (rAAV2-VMD2-hMERTK) Gene Vector to Patients With Retinal Disease Due to MERTK Mutations
Condition: Retinal Disease
Intervention: Genetic: Recombinant Adeno-Associated Virus
15 Active, not recruiting LTFU for Gene Transfer Subjects With Hemophilia B
Condition: Hemophilia B
Intervention:
16 Active, not recruiting Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis
Conditions: Inherited Retinal Dystrophy Due to RPE65 Mutations;   Leber Congenital Amaurosis
Intervention: Biological: AAV2-hRPE65v2
17 Active, not recruiting Choroideremia Gene Therapy Clinical Trial
Condition: Choroideremia
Intervention: Genetic: Injection of AAV2-REP1 (10e11 vg)
18 Completed Safety Study of RPE65 Gene Therapy to Treat Leber Congenital Amaurosis
Condition: Retinal Degeneration
Intervention: Biological: tgAAG76 (rAAV 2/2.hRPE65p.hRPE65)
19 Not yet recruiting Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGA3 Achromatopsia
Condition: Achromatopsia
Intervention: Biological: AGTC-402
20 Recruiting Safety Study of a Gene Transfer Vector (Rh.10) for Children With Late Infantile Neuronal Ceroid Lipofuscinosis
Conditions: Batten Disease;   Late-Infantile Neuronal Ceroid Lipofuscinosis
Interventions: Genetic: AAVrh.10CUhCLN2 vector 9.0x10^11 genome copies;   Genetic: AAVrh.10CUhCLN2 vector 2.85x10^11 genome copies

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