08-2026
|
Immunomodulation Approaches to Improve Safety And Efficacy of Gene Therapy Treatment in Friedreich#s Ataxia
Immunomodulation Approaches to Improve Safety And Efficacy of Gene Therapy Treatment in Friedreich#s Ataxia
|
06-2026
|
CMS Newborn Screening - COQWX
CMS Newborn Screening - COQWX
|
01-2026
|
A Phase 2 Study of the Safety, Efficacy, and Pharmacodynamics of RTA 408 in the Treatment of Friedreich#s Ataxia
A Phase 2 Study of the Safety, Efficacy, and Pharmacodynamics of RTA 408 in the Treatment of Friedreich#s Ataxia
|
12-2025
|
MDA MOVR Data Hub Participation Agreement
MDA MOVR Data Hub Participation Agreement
|
08-2025
|
Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy
Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy
|
08-2025
|
Development of a Novel AAV Vector Capsid Optimized for OA Gene Therapy
Development of a Novel AAV Vector Capsid Optimized for OA Gene Therapy
|
04-2025
|
Control of Breathing and Pompe Disease
Control of Breathing and Pompe Disease
|
06-2024
|
MDA Care Center
MDA Care Center
|
03-2024
|
Preclinical sub-chronic toxicology and biodistribution study of adeno-associated virus (scAAV9-CBh-hAGAopt) mediated gene therapy for Aspartylglucosaminuria Disease in Sprague Dawley rats
Preclinical sub-chronic toxicology and biodistribution study of adeno-associated virus (scAAV9-CBh-hAGAopt) mediated gene therapy for Aspartylglucosaminuria Disease in Sprague Dawley rats
|
11-2023
|
Optimizing AAV Vectors for Central Nervous System transduction
Optimizing AAV Vectors for Central Nervous System transduction
|
08-2023
|
Task Order #10 - Construct Design and Generation Collaboration
Task Order #10 - Construct Design and Generation Collaboration
|
07-2023
|
Cincinnati Zoo Feline and Canine Shedding 2022 Non-GLP
Cincinnati Zoo Feline and Canine Shedding 2022 Non-GLP
|
06-2023
|
SRA - Project Planning
SRA - Project Planning
|
06-2023
|
An Open-Label, Multi-Center, 48-Week Study with a Concurrent Untreated Control Arm to Evaluate the Efficacy and Safety of Eteplirsen in Duchenne Muscular Dystrophy
An Open-Label, Multi-Center, 48-Week Study with a Concurrent Untreated Control Arm to Evaluate the Efficacy and Safety of Eteplirsen in Duchenne Muscular Dystrophy
|
05-2023
|
Immune response to AAV Vector Capsid and Assessment of Eligibility for AAV-mediated Gene Therapy for Duchenne Muscular Dystrophy
Immune response to AAV Vector Capsid and Assessment of Eligibility for AAV-mediated Gene Therapy for Duchenne Muscular Dystrophy
|
04-2023
|
Support of NIH catalyze Award for AAV manufacturing and GLP Toxicology studies
Support of NIH catalyze Award for AAV manufacturing and GLP Toxicology studies
|
02-2023
|
Task Order # 12 Pompe Disease Research Support
Task Order # 12 Pompe Disease Research Support
|
12-2022
|
Sarepta TO #1: Support of PGTC Cores
Sarepta TO #1: Support of PGTC Cores
|
11-2022
|
Development of a novel gene therapy for lupus
Development of a novel gene therapy for lupus
|
07-2022
|
Breathing Research and Therapeutics (BREATHE)
Breathing Research and Therapeutics (BREATHE)
|
06-2022
|
Earl and Christy Powell University Chair in Gene Therapy and Genetics Research - F017885/6
Earl and Christy Powell University Chair in Gene Therapy and Genetics Research - F017885/6
|
06-2022
|
Cure FA Research Foundation
Cure FA Research Foundation
|
05-2022
|
An open-label, multicenter, multinational, extension study of the long-term safety and pharmacokinetics of repeated biweekly infusions of neoGAA in patients with Pompe disease
An open-label, multicenter, multinational, extension study of the long-term safety and pharmacokinetics of repeated biweekly infusions of neoGAA in patients with Pompe disease
|
04-2022
|
A Long-term Study to Evaluate Growth and Development Outcomes in Patients with Infantile-Onset Pompe Disease Who Are Receiving Myzoyme (alglucosidase alfa)
A Long-term Study to Evaluate Growth and Development Outcomes in Patients with Infantile-Onset Pompe Disease Who Are Receiving Myzoyme (alglucosidase alfa)
|
03-2022
|
Expanded access trial of systemic delivery of aspartoacylase (rAAV0-CB6-AspA) gene vector in a single patient with canavan disease
Expanded access trial of systemic delivery of aspartoacylase (rAAV0-CB6-AspA) gene vector in a single patient with canavan disease
|
12-2021
|
TO #1 - Access and Transfer of rAAV9-CMV-hAADC Production and Testing Methods#
TO #1 - Access and Transfer of rAAV9-CMV-hAADC Production and Testing Methods#
|
08-2021
|
An Open-Label, Multi-Center Study to Evaluate the Safety, Efficacy and Tolerability of Eteplirsen in Early-Stage Duchenne Muscular Dystrophy
An Open-Label, Multi-Center Study to Evaluate the Safety, Efficacy and Tolerability of Eteplirsen in Early-Stage Duchenne Muscular Dystrophy
|
07-2021
|
Phase II Study of AAV9-GAA Gene Transfer in Pompe Disease
Phase II Study of AAV9-GAA Gene Transfer in Pompe Disease
|
07-2021
|
Failed Regeneration in the Muscular Dystrophies: Inflammation, Fibrosis and Fat
Failed Regeneration in the Muscular Dystrophies: Inflammation, Fibrosis and Fat
|
07-2021
|
Task Order #4 - Assay Development Support for Friedreich#s Ataxia
Task Order #4 - Assay Development Support for Friedreich#s Ataxia
|
05-2021
|
Determination of anti-AAV9 IgG & anti-AAV9 IgM level in mouse serum samples
Determination of anti-AAV9 IgG & anti-AAV9 IgM level in mouse serum samples
|
05-2021
|
Data Registry Services
Data Registry Services
|
03-2021
|
GLP Tissue and Shedding Biodistribution Study in Nonhuman Primates
GLP Tissue and Shedding Biodistribution Study in Nonhuman Primates
|
12-2020
|
Analysis of Cincinnati Zoo Feline Shedding
Analysis of Cincinnati Zoo Feline Shedding
|
12-2020
|
Non-GLP Expression Study in C57BL/6 Mice
Non-GLP Expression Study in C57BL/6 Mice
|
12-2020
|
Analysis of Human Samples for Biodistribution and Circulating Antibodies
Analysis of Human Samples for Biodistribution and Circulating Antibodies
|
12-2020
|
Sarepta Task Order #7 - Pompe Canine Colony Housing Program
Sarepta Task Order #7 - Pompe Canine Colony Housing Program
|
12-2020
|
Fox Research Fund
Fox Research Fund
|
12-2020
|
Sunshine Children's Fund
Sunshine Children's Fund
|
12-2020
|
Earl and Christy Powell University Chair in Gene Therapy and Genetics Research -
Earl and Christy Powell University Chair in Gene Therapy and Genetics Research -
|
09-2020
|
Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy
Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy
|
08-2020
|
UFRCC for Cardiovascular Cell Therapy Research Network
UFRCC for Cardiovascular Cell Therapy Research Network
|
06-2020
|
Solid TO #15 Evaluation of Immune Management in AAV-?Dys Gene Therapy
Solid TO #15 Evaluation of Immune Management in AAV-?Dys Gene Therapy
|
04-2020
|
Antisense oligonucleotide therapy for a novel humanized GAA rat model of Pompe Disease
Antisense oligonucleotide therapy for a novel humanized GAA rat model of Pompe Disease
|
12-2019
|
Task Order #13 - Evaluation of SGT-01 in Neonatal NHP using Immune Modulation
Task Order #13 - Evaluation of SGT-01 in Neonatal NHP using Immune Modulation
|
12-2019
|
GLP Tissue-only Biodistribution Study in Nonhuman Primates
GLP Tissue-only Biodistribution Study in Nonhuman Primates
|
12-2019
|
Solid TO#12 - Study ID:UFGTC15-007/ UFGTC16-007/UFGTC16-008
Solid TO#12 - Study ID:UFGTC15-007/ UFGTC16-007/UFGTC16-008
|
12-2019
|
Neuromuscular Disease/Clinical Trial Support Fund
Neuromuscular Disease/Clinical Trial Support Fund
|
11-2019
|
Process Development for AAV Manufacturing of AAV-CB-hFXN
Process Development for AAV Manufacturing of AAV-CB-hFXN
|
06-2019
|
Respiratory Muscle Function in Untreated X-linked Myotubular Myopathy
Respiratory Muscle Function in Untreated X-linked Myotubular Myopathy
|
03-2019
|
Toxicology Study of BS01 in Rd1 mice
Toxicology Study of BS01 in Rd1 mice
|
03-2019
|
OneFlorida Clinical Research Consortium Phase II
OneFlorida Clinical Research Consortium Phase II
|
03-2019
|
POM002 LAB SCERVICES AGREEMENT - GAA ACTIVITY ASSAY ANDGLYCOGEN BIOCHEMICAL ASSAY TESTS
POM002 LAB SCERVICES AGREEMENT - GAA ACTIVITY ASSAY ANDGLYCOGEN BIOCHEMICAL ASSAY TESTS
|
01-2019
|
Diaphragm pacing and rehabilitation in Pompe disease
Diaphragm pacing and rehabilitation in Pompe disease
|
12-2018
|
Lacerta Task Order #3 - NHP TAb Screening
Lacerta Task Order #3 - NHP TAb Screening
|
12-2018
|
Limelight TO #1 - Optimizing Hydrodynamic Transduction Injection Procedure
Limelight TO #1 - Optimizing Hydrodynamic Transduction Injection Procedure
|
12-2018
|
Analysis of Cincinnati Zoo Feline Shedding
Analysis of Cincinnati Zoo Feline Shedding
|
12-2018
|
BSF Human Material Storage
BSF Human Material Storage
|
10-2018
|
McGollie - Longitudinal, Observations Study of Subjects with Cardioskeletal Myopathy
McGollie - Longitudinal, Observations Study of Subjects with Cardioskeletal Myopathy
|
08-2018
|
The relationship between genomic variants and MRI/MRS markers in DMD
The relationship between genomic variants and MRI/MRS markers in DMD
|
08-2018
|
SOLID TO #5 ASSESSMENT OF SAFETY/EFFICACY OF IVAAV-MICRO-DYSTROPHIN CONSTRUCT
SOLID TO #5 ASSESSMENT OF SAFETY/EFFICACY OF IVAAV-MICRO-DYSTROPHIN CONSTRUCT
|
08-2018
|
Becker Muscular Dystrophy - A Natural History Study to Predict Efficacy of Exon Skipping
Becker Muscular Dystrophy - A Natural History Study to Predict Efficacy of Exon Skipping
|
07-2018
|
Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease
Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease
|
06-2018
|
Hex-003-004
Hex-003-004
|
06-2018
|
Development of a Strength Training Protocol in Duchenne Muscular Dystrophy
Development of a Strength Training Protocol in Duchenne Muscular Dystrophy
|
06-2018
|
Control of Breathing and Pompe Disease
Control of Breathing and Pompe Disease
|
05-2018
|
Open-Label, Multiple-Dose, Efficacy, Safety, and Tolerability Study of Eteplirsen in Subjects with Duchenne Muscular Dystrophy who Participated in Study 4658-US-201
Open-Label, Multiple-Dose, Efficacy, Safety, and Tolerability Study of Eteplirsen in Subjects with Duchenne Muscular Dystrophy who Participated in Study 4658-US-201
|
04-2018
|
A Study of Respiratory Muscle Strength, including Effort-Independent Measures, in Subjects with Late-Onset Pompe Disease
A Study of Respiratory Muscle Strength, including Effort-Independent Measures, in Subjects with Late-Onset Pompe Disease
|
02-2018
|
A Phase 4, Open-Label, Prospective Study in Patients with Pompe Disease to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale
A Phase 4, Open-Label, Prospective Study in Patients with Pompe Disease to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale
|
02-2018
|
Advanced Gene Therapy for Treatment of Cardiomyopathy and Respiratory Insufficiency in Duchenne Muscular Dystrophy
Advanced Gene Therapy for Treatment of Cardiomyopathy and Respiratory Insufficiency in Duchenne Muscular Dystrophy
|
12-2017
|
A Medical Chart Review of Patients with X-Linked Myotubular Myopathy (XLMTM)
A Medical Chart Review of Patients with X-Linked Myotubular Myopathy (XLMTM)
|
09-2017
|
MD110050 Optical Imaging of Dystrophic and Damaged Muscle
MD110050 Optical Imaging of Dystrophic and Damaged Muscle
|
07-2017
|
Production and Testing of rAAV9-DES-hGAA Clinical Product and rAAV9 Excipient TO#2
Production and Testing of rAAV9-DES-hGAA Clinical Product and rAAV9 Excipient TO#2
|
06-2017
|
Solid Fellow
Solid Fellow
|
06-2017
|
A Pilot Study of the Effects of Oral Administration of Zavesca on Anti-rhGAA Immune Response in Subjects with Pompe Disease Receiving rhGAA Enzyme Replacement Therapy
A Pilot Study of the Effects of Oral Administration of Zavesca on Anti-rhGAA Immune Response in Subjects with Pompe Disease Receiving rhGAA Enzyme Replacement Therapy
|
05-2017
|
Heart and Skeletal Muscle Metabolism, Energetics and Function in Barth Syndrome
Heart and Skeletal Muscle Metabolism, Energetics and Function in Barth Syndrome
|
04-2017
|
A Long Term Study for Extended BMN 701 Treatement of Patients with Pompe Disease who Have Particpated in a BMN701 Study
A Long Term Study for Extended BMN 701 Treatement of Patients with Pompe Disease who Have Particpated in a BMN701 Study
|
03-2017
|
Development therapeutic gene therapy vector for N-glycanase 1-Deficiency
Development therapeutic gene therapy vector for N-glycanase 1-Deficiency
|
02-2017
|
Response to Diaphragmatic Pacing in Subjects with Pompe
Response to Diaphragmatic Pacing in Subjects with Pompe
|
12-2016
|
Project Development Planning
Project Development Planning
|
12-2016
|
Preclinical Study of Adeno-Associated Virus Mediated Gene Therapy in Rat Model of Pompe Disease # Immunomodulation Study
Preclinical Study of Adeno-Associated Virus Mediated Gene Therapy in Rat Model of Pompe Disease # Immunomodulation Study
|
12-2016
|
AAV9 Pompe Rodent Study
AAV9 Pompe Rodent Study
|
11-2016
|
A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) in rhGAA Exposed Subjects with Late-onset Pompe Disease
A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) in rhGAA Exposed Subjects with Late-onset Pompe Disease
|
08-2016
|
Clinical and Translational Science Institute
Clinical and Translational Science Institute
|
08-2016
|
A RANDOMED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PHASE 3 TRIAL OF TADALAFIL FOR DUCHENNE MUSCULAR DYSTROPHY
A RANDOMED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PHASE 3 TRIAL OF TADALAFIL FOR DUCHENNE MUSCULAR DYSTROPHY
|
06-2016
|
Development of Clinical Translational Studies
Development of Clinical Translational Studies
|
05-2016
|
Request for Support to Plan Clinical Gene Therapy Program for BTHS
Request for Support to Plan Clinical Gene Therapy Program for BTHS
|
03-2016
|
Phase I/II Trial of Diaphragm Delivery of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase (rAAV1-CMV-GAA) Gene Vector in Patients with Pompe Disease
Phase I/II Trial of Diaphragm Delivery of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase (rAAV1-CMV-GAA) Gene Vector in Patients with Pompe Disease
|
03-2016
|
Imaging Core: Protocol 701-301
Imaging Core: Protocol 701-301
|
03-2016
|
MP3 Conference Fund
MP3 Conference Fund
|
02-2016
|
Pompe Gene Therapy
Pompe Gene Therapy
|
01-2016
|
AGLU07510 PEAS
AGLU07510 PEAS
|
09-2015
|
A Phase III Double-Blind, Randomised, Placebo-Controlled
A Phase III Double-Blind, Randomised, Placebo-Controlled
|
08-2015
|
Assessment of Cardiopulmonary Function in Duchenne Muscu
Assessment of Cardiopulmonary Function in Duchenne Muscu
|
08-2015
|
Magnetic Resonance Imaging and Biomarkers for Muscular D
Magnetic Resonance Imaging and Biomarkers for Muscular D
|
07-2015
|
Failed Regeneration in the Muscular Dystrophies: Inflamm
Failed Regeneration in the Muscular Dystrophies: Inflamm
|
05-2015
|
Post-doctoral training in glycogen storage disease type
Post-doctoral training in glycogen storage disease type
|
05-2015
|
NEOGAA Clinical Trial
NEOGAA Clinical Trial
|
02-2015
|
Pompe Research - UF Matching Account
Pompe Research - UF Matching Account
|
01-2015
|
Treatment of Pompe Disease via Retrograde Transduction o
Treatment of Pompe Disease via Retrograde Transduction o
|
11-2014
|
AT2220-010: An Open-Label, Multi-Center, International
AT2220-010: An Open-Label, Multi-Center, International
|
10-2014
|
Amicus Data Set Agrmt
Amicus Data Set Agrmt
|
09-2014
|
Upper airway gene therapy in a mouse model of Pompe dise
Upper airway gene therapy in a mouse model of Pompe dise
|
08-2014
|
Spinal and brainstem respiratory neurons in Pompe diseas
Spinal and brainstem respiratory neurons in Pompe diseas
|
08-2014
|
In Vivo Characterization of Novel Modified Recombinant H
In Vivo Characterization of Novel Modified Recombinant H
|
07-2014
|
Failed Regeneration in the Muscular Dystrophies: Inflamm
Failed Regeneration in the Muscular Dystrophies: Inflamm
|
06-2014
|
Gene Therapy for Lung and Cardiovascular Disease
Gene Therapy for Lung and Cardiovascular Disease
|
05-2014
|
Planning a Multi-Center Clinical Trial of PDE5
Planning a Multi-Center Clinical Trial of PDE5
|
05-2014
|
A Phase 4 Prospective Exploratory Muscle Biopsy, Biomark
A Phase 4 Prospective Exploratory Muscle Biopsy, Biomark
|
12-2013
|
Oxyrane
Oxyrane
|
11-2013
|
A PHASE I OPEN-LABEL SAFETY/TOLERABILITY/ PK/PD STUDY OF
A PHASE I OPEN-LABEL SAFETY/TOLERABILITY/ PK/PD STUDY OF
|
09-2013
|
MRI Central Core Lab Services for POM001
MRI Central Core Lab Services for POM001
|
07-2013
|
Failed Regeneration in the Muscular Dystrophies
Failed Regeneration in the Muscular Dystrophies
|
06-2013
|
Phase I/II Trial of Diaphragm Delivery of aAAV1-CMV-GAA
Phase I/II Trial of Diaphragm Delivery of aAAV1-CMV-GAA
|
03-2013
|
Oral immune modulatory therapy using antigens bioencapsu
Oral immune modulatory therapy using antigens bioencapsu
|
01-2013
|
Exercise Video for Pompe patients
Exercise Video for Pompe patients
|
11-2012
|
Chatlos Gene Therapy Research - Companion Award
Chatlos Gene Therapy Research - Companion Award
|
11-2012
|
UFF Companion Award - Gene Therapy Research
UFF Companion Award - Gene Therapy Research
|
09-2012
|
Control of Breathing and Glycogen Storage
Control of Breathing and Glycogen Storage
|
09-2012
|
Gene Therapy for Cardiac and Skeletal Myopathies
Gene Therapy for Cardiac and Skeletal Myopathies
|
08-2012
|
MR Monitoring of PTC124 Treatment in DMD
MR Monitoring of PTC124 Treatment in DMD
|
07-2012
|
Failed Regeneration in the Muscular Dystrophies:
Failed Regeneration in the Muscular Dystrophies:
|
07-2012
|
CEDARS-SINAI MEDICAL CENTER CONSULTING SERVICES AGREEMEN
CEDARS-SINAI MEDICAL CENTER CONSULTING SERVICES AGREEMEN
|
06-2012
|
GENE THERAPY CENTER
GENE THERAPY CENTER
|
06-2012
|
Clinical Trial Support for Muscular Dystrophy Studies
Clinical Trial Support for Muscular Dystrophy Studies
|
06-2012
|
Alglucosidase ALFA Temporary Access Program
Alglucosidase ALFA Temporary Access Program
|
03-2012
|
GILT Tagged Enzyme Replacemetn Therapy for Pompe Disease
GILT Tagged Enzyme Replacemetn Therapy for Pompe Disease
|
02-2012
|
UFCC for Cardiovascular Cell Therapy Research Network
UFCC for Cardiovascular Cell Therapy Research Network
|
02-2012
|
GENE THERAPY IN MOUSE MODEL OF BARTH SYNDROME
GENE THERAPY IN MOUSE MODEL OF BARTH SYNDROME
|
02-2012
|
ENZYME ACTIVITY AND CRIM STATUS IN PATIENTS WITH LATE-ON
ENZYME ACTIVITY AND CRIM STATUS IN PATIENTS WITH LATE-ON
|
12-2011
|
Gene Therapy for Glycogen Storage Diseaes Type Ia Human
Gene Therapy for Glycogen Storage Diseaes Type Ia Human
|
10-2011
|
Failed Regeneration in the Muscular Dystrophies:
Failed Regeneration in the Muscular Dystrophies:
|
07-2011
|
Module I Clinical Trials of Gene Therapy for Leber Conge
Module I Clinical Trials of Gene Therapy for Leber Conge
|
07-2011
|
Recombinant AAV For Correction of Genetic Abnormalities
Recombinant AAV For Correction of Genetic Abnormalities
|
03-2011
|
Barth Syndrome Registry and DNA Bank
Barth Syndrome Registry and DNA Bank
|
10-2010
|
AAV-modified Human Skeletal Myoblast Therapy for Heart F
AAV-modified Human Skeletal Myoblast Therapy for Heart F
|
08-2010
|
Pilot Study to Determine the Efficacy of Newborn Screeni
Pilot Study to Determine the Efficacy of Newborn Screeni
|
07-2010
|
Clinical Trials of Gene Therapy for Leber Congenital Ama
Clinical Trials of Gene Therapy for Leber Congenital Ama
|
07-2010
|
Genzyme Support Services Agreement
Genzyme Support Services Agreement
|
06-2010
|
NGVL Toxicology Laboratory
NGVL Toxicology Laboratory
|
04-2010
|
Magnetic Resonance Imaging and BioMarkers for Muscular D
Magnetic Resonance Imaging and BioMarkers for Muscular D
|
04-2010
|
MRI Assessment of PTC124 Treatment in Boys with DMD
MRI Assessment of PTC124 Treatment in Boys with DMD
|
03-2010
|
An Open-Label, Multicenter Study of Oral AT2220 in Patie
An Open-Label, Multicenter Study of Oral AT2220 in Patie
|
03-2010
|
POM-CL-002 Multienter study to eval new method to measur
POM-CL-002 Multienter study to eval new method to measur
|
02-2010
|
Cardiac and Skeletal Muscle in Barth Syndrome: Evaluat
Cardiac and Skeletal Muscle in Barth Syndrome: Evaluat
|
08-2009
|
Preclinical Study of Adeno-Associated Virus Mediated Gen
Preclinical Study of Adeno-Associated Virus Mediated Gen
|
08-2009
|
NONINVASIVE MONITORING AND TRACKING OF MUSCLE STEM CELLS
NONINVASIVE MONITORING AND TRACKING OF MUSCLE STEM CELLS
|
07-2009
|
Clinical Trials of Gene Therqapy for Leber Congenital Am
Clinical Trials of Gene Therqapy for Leber Congenital Am
|
07-2009
|
Genzyme AGLU03206 Protocol
Genzyme AGLU03206 Protocol
|
05-2009
|
Pharmacological Chaperone Therapy for Pompe Disease
Pharmacological Chaperone Therapy for Pompe Disease
|
03-2009
|
GENE and PHARMACOLOGICAL THERAPIES FOR CYSTIC FIBROSIS PRO
GENE and PHARMACOLOGICAL THERAPIES FOR CYSTIC FIBROSIS PRO
|
08-2008
|
PROJECT 1: GENE THERAPY USING VIRAL VECTORS FOR LUNG AN
PROJECT 1: GENE THERAPY USING VIRAL VECTORS FOR LUNG AN
|
08-2008
|
Multicenter Study: Characterize the Effects of Pharmacol
Multicenter Study: Characterize the Effects of Pharmacol
|
07-2008
|
Clinical Trials of Gene Therqapy for Leber Congenital Am
Clinical Trials of Gene Therqapy for Leber Congenital Am
|
03-2008
|
Gene Therapy for GSD Type 1a
Gene Therapy for GSD Type 1a
|
12-2007
|
Gene therapy Treament of Pompe Disease with rAAV-GAA
Gene therapy Treament of Pompe Disease with rAAV-GAA
|
12-2007
|
Production of AAV1-AT
Production of AAV1-AT
|
08-2007
|
Clinical Trials of Gene Therapy for Leber Congential Ama
Clinical Trials of Gene Therapy for Leber Congential Ama
|
07-2007
|
Gene Therapy for Leber Congenital Amaurosis
Gene Therapy for Leber Congenital Amaurosis
|
06-2007
|
AAV1-AAT Part 1
AAV1-AAT Part 1
|
06-2007
|
AAV1-AAT Part 11
AAV1-AAT Part 11
|
07-2006
|
OPN-LBL MULTICTR STDY OF THE SAF EFFIC PHARMACOKINETEICS
OPN-LBL MULTICTR STDY OF THE SAF EFFIC PHARMACOKINETEICS
|
07-2006
|
AGLU02403 A Long-Term Continuation Study of Patients wi
AGLU02403 A Long-Term Continuation Study of Patients wi
|
06-2006
|
PHYSIOLOGICAL AND FUNCTIONAL ASSESSMENT OF RAAV-MEDICATE
PHYSIOLOGICAL AND FUNCTIONAL ASSESSMENT OF RAAV-MEDICATE
|
06-2006
|
INCREASED INOS AND REACTIVE OXYGEN SPECIES IN DIABETIC
INCREASED INOS AND REACTIVE OXYGEN SPECIES IN DIABETIC
|
04-2006
|
AGLU01602-AN OPEN-LABEL,MULTICENTER,MULTINATIONAL,DOSE-R
AGLU01602-AN OPEN-LABEL,MULTICENTER,MULTINATIONAL,DOSE-R
|
01-2006
|
Gene Therapy for GSD Type 1a
Gene Therapy for GSD Type 1a
|
09-2005
|
PRECLINICAL STUDY OF GENE REPLACEMENT THERAPY IN MUSCULA
PRECLINICAL STUDY OF GENE REPLACEMENT THERAPY IN MUSCULA
|
08-2005
|
NGVL Toxicology Laboratory
NGVL Toxicology Laboratory
|
08-2005
|
Phase I Study of Mini-Dystrophin Gene in AAV
Phase I Study of Mini-Dystrophin Gene in AAV
|
07-2005
|
Gene Therapy for Leber Congenital Amaurosis
Gene Therapy for Leber Congenital Amaurosis
|
07-2005
|
PROJ 3: TRANSDUCTION OF HEPATOCYTES WITH RECOMBINANT AAV
PROJ 3: TRANSDUCTION OF HEPATOCYTES WITH RECOMBINANT AAV
|
07-2005
|
Pfizer Visiting Professorship Program in Cardiovascular
Pfizer Visiting Professorship Program in Cardiovascular
|
02-2004
|
COMPARISON OF TWO RECOMBINANT ADENO-ASSOCIATED VIRUS VEC
COMPARISON OF TWO RECOMBINANT ADENO-ASSOCIATED VIRUS VEC
|
07-2003
|
GLYOCEN STORAGE DISEASE: HEPATIC GENE THERAPY
GLYOCEN STORAGE DISEASE: HEPATIC GENE THERAPY
|
06-2003
|
FUNCTIONAL ASSESSMENT OF ADEN0-ASSOCIATED VIRUS MEDIATED
FUNCTIONAL ASSESSMENT OF ADEN0-ASSOCIATED VIRUS MEDIATED
|
06-2003
|
INHIBITION OF HUMORAL IMMUNITY IN LIVER-TARGETED GENE
INHIBITION OF HUMORAL IMMUNITY IN LIVER-TARGETED GENE
|
03-2003
|
PRECLINICAL STUDIES OF ENZYME REPLACEMENT THERAPY FOR
PRECLINICAL STUDIES OF ENZYME REPLACEMENT THERAPY FOR
|
08-2002
|
PROJECT 2: GENE THERAPY USING VIRAL VECTORS FOR LUNG AN
PROJECT 2: GENE THERAPY USING VIRAL VECTORS FOR LUNG AN
|
08-2002
|
PROJECT 1: GENE THERAPY USING VIRAL VECTORS FOR LUNG AN
PROJECT 1: GENE THERAPY USING VIRAL VECTORS FOR LUNG AN
|
06-2002
|
GENE THERAPY APPLICATIONS FOR AN INHERITED CARDIOMYOPATH
GENE THERAPY APPLICATIONS FOR AN INHERITED CARDIOMYOPATH
|
06-2002
|
PREVENTION OF INHERITED CARDIOMYOPATHY IN A MURING MODEL
PREVENTION OF INHERITED CARDIOMYOPATHY IN A MURING MODEL
|
06-2002
|
PRECLINICAL STUDY FOR GENETIC CORRECTION OF INHERITED
PRECLINICAL STUDY FOR GENETIC CORRECTION OF INHERITED
|
06-2002
|
FUNCTIONAL ASSESSMANT OF ADENO-ASSOCIATED VIRUS MEDIATED
FUNCTIONAL ASSESSMANT OF ADENO-ASSOCIATED VIRUS MEDIATED
|
06-2002
|
EFFECTS OF INCREASED PROSTACYCLIN SYNTHASE EXPRESSION IN
EFFECTS OF INCREASED PROSTACYCLIN SYNTHASE EXPRESSION IN
|
06-2002
|
TREATMENT OF A MOUSE MODEL OF HEMOPHILIA A USING AAV VEC
TREATMENT OF A MOUSE MODEL OF HEMOPHILIA A USING AAV VEC
|
04-2002
|
PRECLINICAL STUDY OF PARVOVIRUS MEDIATED GENE THERAPY
PRECLINICAL STUDY OF PARVOVIRUS MEDIATED GENE THERAPY
|
01-2002
|
INDUSTION OF CARDIAC AND ENDITHELIAL ALLOGRAFT TOLERANCE
INDUSTION OF CARDIAC AND ENDITHELIAL ALLOGRAFT TOLERANCE
|
06-2001
|
RESEARCH RESOURCES PROGRAM FOR MEDICAL SCHOOLS
RESEARCH RESOURCES PROGRAM FOR MEDICAL SCHOOLS
|
03-2001
|
CARDIAC MUSCLE REGENERATION USING MESENCHYMAL STEM CELLS
CARDIAC MUSCLE REGENERATION USING MESENCHYMAL STEM CELLS
|
12-2000
|
CORRECTION OF INHERITED CARDIOMYOPATHY USING AAV VECTORS
CORRECTION OF INHERITED CARDIOMYOPATHY USING AAV VECTORS
|
11-2000
|
SCALEABLE PRODUCTION OF RAAV VECTORS
SCALEABLE PRODUCTION OF RAAV VECTORS
|
10-2000
|
RESEARCH RESOURCES PROGRAM FOR MEDICAL SCHOOLS
RESEARCH RESOURCES PROGRAM FOR MEDICAL SCHOOLS
|
09-2000
|
NEOCYTOLYSIS: MECHANISMS and LIMITATIONS
NEOCYTOLYSIS: MECHANISMS and LIMITATIONS
|
06-2000
|
CORRECTION OF INHERITED CARDIOMYOPATHY IN A MURINE EMBRY
CORRECTION OF INHERITED CARDIOMYOPATHY IN A MURINE EMBRY
|
06-2000
|
TREATMENT OF MOUSE MODEL OF HEMOPHILIA A USING AAV VECTO
TREATMENT OF MOUSE MODEL OF HEMOPHILIA A USING AAV VECTO
|
05-1999
|
GENE REPLACEMENT IN EMBRYONIC MODELS OF ACID MALTASE
GENE REPLACEMENT IN EMBRYONIC MODELS OF ACID MALTASE
|