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Reports



# Title
1 Single Dose 6-Month Toxicity Study ofAdeno-Associated Virus-Cystic Fibrosis (AAV-CFTR) Gene Vector in the Rhesus Monkey
2 Single Dose Subcutaneous Biodistribution Study of Recombinant Adenovirus Expressing PDGF-B in C57B1/6 Mice
3 60-Day Subcutaneous Toxicity Study of Recombinant Adenovirus Expressing PDGF-B in C57B1/6 Mice
4 Safety and Germline Transmission of rAAV2-hAAT Vector After Intramuscular Injection in the Baboon
5 Toxicology and Biodistribution Study of rAAV-AAT Vectors in Rabbit Tissues
6 Single Dose 3-month Toxicity Study of Adeno-Associated Virus AAT Gene Vector in the C57/BL6 Mouse
7 Toxicity and Vector Distribution Study in Rats Following A Single Injection into the Submandibular Duct. Test article Adenovirus/AdCMVH3
8 Single Dose Biodistribution Study of AAV-CB-AAT Comparing IM and IV Routes of Vector Administration in the C57/B16 Mouse
9 A 12-week Toxicity Study of DNA Vaccine (pTVG-HP) Encoding Human Prostatic Acid Phosphatase (PAP) Administered Intradermally to Male Lewis Rats
10 Evaluation of Potential Toxicity of Electroporation Mediated Delivery of a Plasmid Encoding for IL-12 in a Mouse Melanoma Model
11 Toxicology and Biodistribution Study of rAAV-1-CB-hAAT in New Zealand White Rabbits
12 Effect of HCV Infection in Safety and Efficacy of Liver Delivery of rAAV-AAT
13 Toxicology and Biodistribution Study of AAV-Mediated Gene Therapy for Muscular Dystrophy
14 Toxicological Safety Evaluation of DNA Plasmid Vaccines against HIV-1, Ebola, Severe Acute Respiratory Syndrome, or West Nile Virus Is Similar Despite Differing Plasmid Backbones or Gene-Inserts
15 28-Day Prechronic Toxicity, Biodistribution and Transgene Expression Study in Fischer 344 Rats for Single Submandibular Gland Injections of an Adenoviral Vector Carrying the Human Growth Hormone Gene (Ad-hGH)
16 Dose Response of H5.001RSVTK in C57BL/6 Female Mice after Intraperitoneal Inoculation, with Ganciclovir on Days 2 to 15 Delivered Intraperitoneally (Ovarian Cancer Project)
17 Biodistribution of DNA Plasmid Vaccines against HIV-1, Ebola, Severe Acute Respiratory Syndrome, or West Nile Virus Is Similar, without Integration, despite Differing Plasmid Backbones or Gene Inserts
18 Myocarditis Following Adeno-Associated Viral Gene Expression of Human Soluble TNF Receptor (TNFRII.Fc) in Baboon Hearts.
19 A Single-Dose Toxicology Study of NP2 Administered Subcutaneously in BALB/c Mice with 90 Day Follow-up
20 A Single-Dose 90 Day Biodistribution Study of Viral Test Article NP2 Administered Subcutaneously in BALB/C Mice
21 Three Month Toxicity and Biodistribution Study of AAV2-SB-hRPE65 Following a Single Subretinal or Intravitreal Injection in Cynomolgus Monkeys.
22 Toxicology Study of rAAV-1-CB-hAAT Summary of Findings
23 Biodistribution Study of rAAV-1-CB-hAAT Summary of Findings
24 One-week Toxicity and Biodistribution Study of AAV2-SB-hRPE65 Following a Single Subretinal Injection in Cynomolgus Monkeys.
25 RPE65 Canine Pre-Clinical Studies
26 Toxicology and Biodistribution Study of Adenylyl Cyclase VI Gene Transfer for Congestive Heart Failure Summary of Findings
27 Toxicity and Biodistribution Report of rAAV6-CMV-hSERCA2a Following Injection into the Heart in Canines.
28 A 90-Day Toxicology and Biodistribution Study of Viral Test Article NC3 Administered Intracranially in BALB/c Mice
29 Preclinical Study of Adeno-Associated Virus Mediated Gene Therapy for Pompe Disease (Study ID# GSDII-001)
30 Preclinical Study of Adeno-Associated Virus Mediated Gene Therapy for Pompe Disease by direct injection into the diaphragm of New Zealand White Rabbits (Study ID # GSDII-002)
31 Direct Comparative Analysis of rAAV1 and rAAV5 Pseudotyped Vectors Using Aerosol Delivery of Firefly and Renila Luciferase Reporters Co-delivered to the Lungs of Chimpanzees.
32 Biodistribution Report of rAAV6-CMV-SERCA2a Following Injection into the Heart in Canines
33 Biodistribution and Toxicological Safety of Adenovirus Type 5 and Type 35 Vector Vaccines Against Human Immunodeficiency Virus-1 (HIV-1), Ebola, or Marburg Are Similar Despite Differing Adenovirus Serotype Vector, Manufacturer's Construct, or Gene Inserts.
34 Preclinical Study of Adeno-Associated Virus Mediated Gene Therapy for Retinal Dystrophy (AAV2-VMD2-hMerTK)
35 Toxicity Study of rAAV1 CB-hAAT (TFX) and rAAV1 CB hAAT (HSV) Administered Intramuscularly in the C57BL/6 Mice
36 Assessment of Safety and Tissue Distribution of the Lenti/BAS3-FB Lentiviral Vector in C57BL/6J Mice
37 Pharmacology/Toxicology Study of the EFS-ADA Lentiviral Vector in Mice
38 Assessment of Toxicity and Biodistribution of the AAV2/8LSPhGAApA
39 Toxicology and Biodistribution of rAAV1-CB-hAAT Administered in Male and Female Rhesus Macaques by Isolated Limb Infusion
40 Single Dose Toxicity Study of sc-rAA V2.5IL-1Ra in a Mono-iodoacetate-Induced Experimental Model of Osteoarthritis in Wistar Rats
41 Therapeutic rAAVrh10 Mediated SOD1 Silencing in Adult SOD1G93A Mice and Nonhuman Primates
42 Gene Therapy Fully Restores Vision to the All-Cone Nrl - / -Gucy2e- / - Mouse Model of Leber Congenital Amaurosis
43 Progress with Recombinant Adeno-Associated Virus Vectors for Gene Therapy of Alpha-1 Antitrypsin Deficiency
44 NOVEL VECTOR DESIGN AND HEXOSAMINIDASE VARIANT ENABLING SELF- COMPLEMENTARY AAV FOR THE TREATMENT OF TAY-SACHS DISEASE
45 Systemic Gene Transfer of a Hexosaminidase Variant Using an scAAV9.47 Vector Corrects GM2 Gangliosidosis in Sandhoff Mice
46 In Vivo CRISPR/Cas9 Gene Editing Corrects Retinal Dystrophy in the S334ter-3 Rat Model of Autosomal Dominant Retinitis Pigmentosa.
47 A regulatable AAV vector mediating GDNF biological effects at clinically-approved sub-antimicrobial doxycycline doses
48 Preclinical Development of New Therapy for Glycogen Storage Diseases
49 Final Preclinical Development of Gene Therapy for Post-operative Atrial Fibrillation
50 Effects of Co-Administration of NY-ESO-1 TCR genetically modified T cells and Hematopoietic Stem Cells (HSCs) in HLAA2.1/Kb mice
51 Assessment of the Safety Biodistribution of rAAV6 BARKct Administered Once in Male and Female Sprague Dawley Rats
52 Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype