Skip to main content
Patient Education Logo
Home > Gene & Cell Therapy 101 > Gene Therapy Centers

Gene Therapy Centers

This list of gene therapy centers in the U.S. is intended for use by patients, advocates, and caregivers who are seeking gene therapy research for a rare disease. This list excludes oncology (cancer) due to the wide scope of cancers and number of centers working on them. The Blood & Marrow Transplant Information Network has a list of medical centers offering CAR T-cell therapy. If you are unsure whether a clinical trial is open for your disease, search by diagnosis using the ASGCT Clinical Trials Finder. To suggest a center or make an update to a center listed please contact us.



Disease focus area* 




Boston Children’s Hospital Gene Therapy Program 

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 

Boston Children’s Hospital  

300 Longwood Avenue, Boston MA 02115 


Cell and Gene Therapy Collaborative at CHOP 

Beta thalassemia 



Leber congenital amaurosis (LCA) 

Methylmalonic acidemia  

Mucopolysaccharidosis (MPS I, MPS II)  

Phenylketonurias (PKU) 

Sickle cell disease 

Spinal muscular atrophy (SMA) 


The Children’s Hospital of Philadelphia 

3501 Civic Center Blvd 

Philadelphia, PA 19104 


Learn more 

Cell and Gene Therapy Center at RowanSOM / Cooper Hospital 

Canavan disease 

Neurological disorders 


Rowan University, School of Osteopathic Medicine 

2 Medical Center Drive, Science Center #253 

Stratford, NJ 08084 


P: (856) 566-6334 


Learn more 




Center for Cell and Gene Therapy at Baylor College of Medicine 


Cardiovascular disease 


Baylor College of Medicine 
One Baylor Plaza, Rm N1002 
Houston, TX 77030 

P: (832) 824-4670 


Learn more 




Center for Cell Engineering at Memorial Sloan Kettering Cancer Center 

Blood disorders 

Neurologic diseases 

Center for Cell Engineering 
Memorial Sloan Kettering Cancer Center, Box 182 
1275 York Avenue 
New York, NY 10065 


P: (212) 639-6394 


Department of Genetics and Genomic Sciences at Mount Sinai 

Alzheimer's disease 

Cardiovascular illness 

Crohn's disease 


Infectious diseases 

Lysosomal storage disorders 

Psychiatric illnesses 

Ulcerative colitis 


One Gustave L. Levy Place 
Box 1498 
New York, NY 10029 

P: (212) 659-8542 


Learn more 

Department of Microbiology & Molecular Genetics 

Cystic fibrosis 

Cardiovascular disease 

University of Pittsburgh 

523 Bridgeside Point II 

450 Technology Drive 

Pittsburgh, PA 15219 


P: (412) 648-9570 


Learn more 

Gene Therapy Program at UPenn 

Blood diseases  

Eye disorders 

Infectious diseases 

Lung diseases 

Metabolic diseases 

Neurologic diseases 

Neuromuscular disorders 

Renal/liver diseases 


Suite 1200 Translational Research Labs (TRL) 

125 S. 31st Street 

Philadelphia, PA 19104 

P: (215) 898-1281 


Learn more 


Grousbeck Gene Therapy Center 



Neurosensory disorders 


Gene Delivery Technology Development 



20 Stanford Street 

Boston, MA 02114 


P: (617)-573-6307 

Email direct 

Email admin  

Learn more 


Horae Gene Therapy Center 

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  


UMass Medical School  

368 Plantation Street Worcester, MA 01605 

P: (508) 856-3394 


Learn more 


IU School of Medicine Brown Center for Immunotherapy  

Alzheimer’s disease 


Walther Hall 
980 W. Walnut Street 
Indianapolis, IN 46202 

P: (317) 278-6969 

Learn more 


Mathews Center for Cellular Therapy  

Adult stem cell transplant  

Allergy and immunology 

Solid organ transplant  


Northwestern Memorial Hospital  

710 N. Fairbanks Court  

Rm 7-613 

Chicago, IL 60611 


P: (312) 926-2210 


Learn more 

Nationwide Children’s Center for Gene Therapy   

Infectious Diseases 


Lysosomal storage disorders 

Neuromuscular disorders 

Neurodegenerative diseases 

Neonatal hypertension 

Reperfusion injury 



The Abigail Wexner Research Institute at Nationwide Children's Hospital 

Research Bldg. II 

Columbus, OH 43205 

P: (614) 722-5615 


Learn more 

Nemours Children’s Hospital - Central Florida 

  • Duchenne Muscular Dystrophy
  • Spinal muscular atrophy (SMA)

Nemours Children’s Hospital

6535 Nemours Parkway, Orlando Florida 32827

(407) 650-7715

Powell Gene Therapy Center 


Powell Center for Rare Disease Research  


Cardiovascular diseases 

Central nervous system disorders 

Endocrine disorders 

Eye diseases 

Inflammatory disorders 

Metabolic disorders 

Neuromuscular disorders 

Pulmonary diseases 


University of Florida 

Academic Research Building College of Medicine 

1200 Newell Drive 

Gainesville, FL 32610 

Gene Therapy Center 

P: (352) 294-5171 



Center for Rare Disease Research 


The Vaccine and Gene Therapy Institute at OHSU 


Chronic viral infection-associated diseases 

Infectious diseases 

Infectious diseases of the elderly 

Newly emerging viral diseases 


Oregon Health & Science University – West Campus 

505 N.W. 185th Avenue 

Beaverton, OR 97006 


P: (503) 418-2700  


Learn more 

Tulane Center for Stem Cell Research & Regenerative Medicine 

New therapies for common diseases including stroke, and multiple sclerosis 

Tulane University School of Medicine 

1430 Tulane Avenue 

New Orleans, LA 70112 


P: 504-988-5800 

Learn more 

UNC Gene Therapy Center 


Eye disorders 


Muscular dystrophies 

Neurological disorders (epilepsy) 

Neuropathic lysosomal storage diseases (MPS and Krabbe disease) 


University of North Carolina 

7119 Thurston Bowles Bldg. 

104 Manning Drive 

Campus Box 7352 

Chapel Hill, NC 27599 

P: 919-962-3285 


Learn more 

University of Iowa Gene Therapy Center  

Cystic fibrosis 

Central nervous system disorders 

Diseases affecting muscle, 

vessels, skin, the liver and the lungs 


Roy J. and Lucille A. Carver College of Medicine 

451 Newton Road 

200 Medicine Admin. Building 

Iowa City, IA 52242 

Learn more 

University of Minnesota Gene Therapy Center 

Mucopolysaccharidoses (MPS) and related diseases 

Medical School Department of Pediatrics 

2450 Riverside Avenue 

6th Floor, East Building 

Delivery Code 8952A 

Minneapolis, MN 55454 


P: (612) 624-3113 



UT Southwestern Medical Center  

Central nervous system disorders 

Neurodegenerative diseases 

Neuromuscular disorders 

Lysosomal storage disorders 

Metabolic disorders 

Genetic epilepsies 

Neurodevelopmental disorders 


UT Southwestern Medical Center 

5323 Harry Hines Blvd. 

Dallas, TX 75390 


Learn more 

Last Updated: 07/01/2024

This site uses cookies to offer you a better user experience and to analyze site traffic. By continuing to use this website, you accept our use of cookies.