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Clinical Indication:
Venous Leg Ulcer
Bone Marrow Transplant candidates
CHF
Congestive Heart Failure
Pompe Disease
Multiple
Atrial Fibrillation
Tay Sachs
Sandhoff Disease
Alpha-1 Antitrypsin Deficiency
Muscular Dystrophy
Leber Congenital Amaurosis-1
Cystic Fibrosis
Parkinson Disease
HIV
diabetes
Cancer
Retinal Degenerative Diseases
Heart Failure
Hepatitis C Virus
Sickle Cell Disease and beta-thalassemia
Retinal Disease
ADA-SCID
Amyotrophic Laterial Sclerosis (ALS
Osteoarthritis (OA)
Transgene:
tMCK-h-alphaSG
AAT
CFTR
IL-1Ra
RPE65
SERCA2a
PDGF-B
pTVG-HP
GAApA
HEXA and HEXM
GUCY2D
RSV TK
HLA-A2.1/Kb transgenic mice
hAAT
hGAA
BetaARKct
NUREL C
hGH
Green fluorescent protein (GFP) and HEXM
hACVI
globin
hAAT/bAAT
TNFRII.Fc
hRPE65
hADA cDNA
NUREL P2
hMERTK
KCNH2-G628S (AdKCNH2G628S)
Luciferase (LUC) and human GDNF (hGDNF) cDNA.
human preproenkephalin
SOD1
Vector System:
AAV
HSV
Cas9/gRNA Plasmid
AAV, Ad, LV
Lentivirus and Retrovirus
rAAV6-CMVenh-MLC260- BetaARKct
Adenovirus
Lenti,
Adenovirus AdKCNH2G628S
scAAV
Lenti
DNA plasmids
DNA vaccines
scAAV9/47
#
Title
1
Single Dose 6-Month Toxicity Study of Adeno-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-1
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 G M2 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 and Biodistribution of rAAV6-BARKct Administered Once in Male and Female Sprague Dawley Rats
52
Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype