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Center
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Disease focus area*
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Location
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Contact
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Boston Children’s Hospital Gene Therapy Program
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adrenoleukodystrophy (ALD)
beta thalassemia
hemophilia A and B
inherited retinal disorders
ornithine transcarbamylase (OTC) deficiency
sickle cell disease
spinal muscular atrophy (SMA)
Wiskott-Aldrich syndrome
X-linked severe combined immunodeficiency (SCID-X1)
X-linked chronic granulomatous disease (X-CGD)
relapsed or treatment-resistant B-cell acute lymphoblastic leukemia (ALL)
relapsed or refractory large B-cell lymphoma
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Boston Children’s Hospital
300 Longwood Avenue, Boston MA 02115
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617-919-1992
https://www.childrenshospital.org/programs/gene-therapy-program
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Cell and Gene Therapy Collaborative at CHOP
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Beta thalassemia
Choroideremia
Hemophilia
Leber congenital amaurosis (LCA)
Methylmalonic acidemia
Mucopolysaccharidosis (MPS I, MPS II)
Phenylketonurias (PKU)
Sickle cell disease
Spinal muscular atrophy (SMA)
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The Children’s Hospital of Philadelphia
3501 Civic Center Blvd
Philadelphia, PA 19104
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Contact
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Cell and Gene Therapy Center at RowanSOM / Cooper Hospital
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Canavan disease
Neurological disorders
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Rowan University, School of Osteopathic Medicine
2 Medical Center Drive, Science Center #253
Stratford, NJ 08084
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P: (856) 566-6334
Email
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Center for Cell and Gene Therapy at Baylor College of Medicine
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AIDS
Cardiovascular disease
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Baylor College of Medicine
One Baylor Plaza, Rm N1002
Houston, TX 77030
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P: (832) 824-4670
Email
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Center for Cell Engineering at Memorial Sloan Kettering Cancer Center
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Blood disorders
Neurologic diseases
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Center for Cell Engineering
Memorial Sloan Kettering Cancer Center, Box 182
1275 York Avenue
New York, NY 10065
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P: (212) 639-6394
Email: becharde@mskcc.org
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Department of Genetics and Genomic Sciences at Mount Sinai
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Alzheimer's disease
Cardiovascular illness
Crohn's disease
Diabetes
Infectious diseases
Lysosomal storage disorders
Psychiatric illnesses
Ulcerative colitis
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One Gustave L. Levy Place
Box 1498
New York, NY 10029
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P: (212) 659-8542
Email
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Department of Microbiology & Molecular Genetics
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Cystic fibrosis
Cardiovascular disease
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University of Pittsburgh
523 Bridgeside Point II
450 Technology Drive
Pittsburgh, PA 15219
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P: (412) 648-9570
Email
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Gene Therapy Program at UPenn
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Blood diseases
Eye disorders
Infectious diseases
Lung diseases
Metabolic diseases
Neurologic diseases
Neuromuscular disorders
Renal/liver diseases
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Suite 1200 Translational Research Labs (TRL)
125 S. 31st Street
Philadelphia, PA 19104
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P: (215) 898-1281
Email
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Grousbeck Gene Therapy Center
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Blindness
Deafness
Neurosensory disorders
Other
Gene Delivery Technology Development
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MEE/SERI – 2W
20 Stanford Street
Boston, MA 02114
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P: (617)-573-6307
Email direct
Email admin
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Horae Gene Therapy Center
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Alpha-1 Antitrypsin Deficiency
Canavan disease
Huntington's disease
Inherited and acquired cardiac arrhythmias
Leber congenital amaurosis (LCA)
Lou Gehrig's disease (ALS)
Neurofibromatosis Type 2
Retinitis pigmentosa
Stargardt disease
Tay-Sachs and Sandhoff disease
VLCAD Deficiency
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UMass Medical School
368 Plantation Street Worcester, MA 01605
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P: (508) 856-3394
Email
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IU School of Medicine Brown Center for Immunotherapy
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Alzheimer’s disease
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Walther Hall
980 W. Walnut Street
Indianapolis, IN 46202
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P: (317) 278-6969
Learn more
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Mathews Center for Cellular Therapy
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Adult stem cell transplant
Allergy and immunology
Solid organ transplant
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Northwestern Memorial Hospital
710 N. Fairbanks Court
Rm 7-613
Chicago, IL 60611
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P: (312) 926-2210
Email
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Nationwide Children’s Center for Gene Therapy
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Infectious Diseases
Ischemia
Lysosomal storage disorders
Neuromuscular disorders
Neurodegenerative diseases
Neonatal hypertension
Reperfusion injury
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The Abigail Wexner Research Institute at Nationwide Children's Hospital
Research Bldg. II
Columbus, OH 43205
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P: (614) 722-5615
Email
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Nemours Children’s Hospital - Central Florida
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- Duchenne Muscular Dystrophy
- Spinal muscular atrophy (SMA)
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Nemours Children’s Hospital
6535 Nemours Parkway, Orlando Florida 32827
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(407) 650-7715
https://www.nemours.org/services/neuromuscular-disorders.html
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Powell Gene Therapy Center
and
Powell Center for Rare Disease Research
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Cardiovascular diseases
Central nervous system disorders
Endocrine disorders
Eye diseases
Inflammatory disorders
Metabolic disorders
Neuromuscular disorders
Pulmonary diseases
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University of Florida
Academic Research Building College of Medicine
1200 Newell Drive
Gainesville, FL 32610
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Gene Therapy Center
P: (352) 294-5171
Email
Center for Rare Disease Research
Email
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The Vaccine and Gene Therapy Institute at OHSU
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AIDS
Chronic viral infection-associated diseases
Infectious diseases
Infectious diseases of the elderly
Newly emerging viral diseases
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Oregon Health & Science University – West Campus
505 N.W. 185th Avenue
Beaverton, OR 97006
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P: (503) 418-2700
Email
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Tulane Center for Stem Cell Research & Regenerative Medicine
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New therapies for common diseases including stroke, and multiple sclerosis
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Tulane University School of Medicine
1430 Tulane Avenue
New Orleans, LA 70112
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P: 504-988-5800
Learn more
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UNC Gene Therapy Center
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Eye disorders
Hemophilia
Muscular dystrophies
Neurological disorders (epilepsy)
Neuropathic lysosomal storage diseases (MPS and Krabbe disease)
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University of North Carolina
7119 Thurston Bowles Bldg.
104 Manning Drive
Campus Box 7352
Chapel Hill, NC 27599
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P: 919-962-3285
Email
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University of Iowa Gene Therapy Center
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Cystic fibrosis
Central nervous system disorders
Diseases affecting muscle,
vessels, skin, the liver and the lungs
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Roy J. and Lucille A. Carver College of Medicine
451 Newton Road
200 Medicine Admin. Building
Iowa City, IA 52242
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Learn more
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University of Minnesota Gene Therapy Center
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Mucopolysaccharidoses (MPS) and related diseases
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Medical School Department of Pediatrics
2450 Riverside Avenue
6th Floor, East Building
Delivery Code 8952A
Minneapolis, MN 55454
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P: (612) 624-3113
Email
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| Utah Program for Inherited Neuromuscular Disorders |
Collagen VI myopathies
Duchenne muscular dystrophy
Leukodystrophies
Spinal Muscular Atrophy
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University of Utah- Eccles Institute of Human Genetics; 15 N. 2030 E.
Salt Lake City, UT 84112
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P: 801-585-9717
Email
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UT Southwestern Medical Center
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Central nervous system disorders
Neurodegenerative diseases
Neuromuscular disorders
Lysosomal storage disorders
Metabolic disorders
Genetic epilepsies
Neurodevelopmental disorders
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UT Southwestern Medical Center
5323 Harry Hines Blvd.
Dallas, TX 75390
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Email
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