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42 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 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
3 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
4 Recruiting Batten CLN6 Gene Therapy
Conditions: Batten Disease;   CLN6
Intervention: Drug: scAVV9.CB.CLN6
5 Recruiting RGX-314 Gene Therapy for Neovascular AMD Trial
Conditions: Neovascular Age-related Macular Degeneration;   Wet Age-related Macular Degeneration
Intervention: Biological: RGX-314
6 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
7 Recruiting A Gene Transfer Study for Hemophilia A
Condition: Hemophilia A
Intervention: Genetic: SPK-8011
8 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;   Device: CliniMacs
9 Recruiting Follow-Up Study of Subjects Previously Enrolled in Poxviral Vector Gene Transfer Studies
Condition: Vaccine
Intervention:
10 Recruiting A Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa
Condition: X-Linked Retinitis Pigmentosa
Intervention: Biological: AAV-RPGR
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 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
13 Recruiting E7 TCR T Cells With or Without PD-1 Blockade for Human Papillomavirus-Associated Cancers
Conditions: Cervical Cancer;   Vaginal Cancer;   Anal Cancer;   Penile Cancer;   Oropharyngeal Cancer
Interventions: Biological: E7 TCR Transduced PBL cells;   Drug: pembrolizumab;   Drug: aldesleukin;   Drug: fludarabine;   Drug: cyclophosphamide
14 Not yet recruiting B7.1/IL-2 Leukaemia Cell Vaccine for Non-Transplant AML RFUSIN2-AML2 (NTX)
Conditions: Leukemia, Myeloid, Acute;   Myelodysplastic Syndromes
Intervention: Biological: AML Cell Vaccine
15 Recruiting Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II
Condition: Mucopolysaccharidosis II
Intervention: Biological: SB-913
16 Recruiting A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Subjects With Transfusion-Dependent β-Thalassemia
Condition: Beta-Thalassemia
Intervention: Genetic: LentiGlobin BB305 Drug Product
17 Recruiting AAV2-GDNF for Advanced Parkinson s Disease
Condition: Parkinson's Disease
Intervention: Genetic: Convection enhanced delivery/AAV2-GDNF
18 Recruiting Bone Marrow for Hemoglobinopathy Research
Conditions: Sickle Cell Anemia;   Thalassemia
Intervention:
19 Recruiting T Cell Receptor Immunotherapy Targeting MAGE-A3 for Patients With Metastatic Cancer Who Are HLA-DP0401 Positive
Condition: Metastatic Cancer That Express the MAGE-A3-DP4 Antigen
Interventions: Biological: Anti-MAGE-A3-DP4 TCR;   Drug: Cyclophosphamide;   Drug: Fludarabine;   Drug: Aldesleukin
20 Recruiting Use of G-CSF to Obtain Blood Cell Precursors
Condition: Chronic Granulomatous Disease
Intervention:

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