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Lab and Diseases

UT Southwestern: Gene Therapy Research Labs

The pre-clinical researchers work closely with pediatric neurologists and other disease experts at UT Southwestern to fully understand the clinical implications of gene therapy in the context of the specific disease. This multidisciplinary approach ensures the efficiency and validity of the preclinical research.

Select Diseases Under Study/Disease groups

The UT Southwestern Gene Therapy Program mainly focuses on neurodegenerative, neurodevelopmental, and genetic epilepsy disorders. The following lists select diseases which have ongoing research programs in various stages of preclinical development and translational research:

Disease Program: Disease (Gene)Key Preclinical
UTSW Investigators
Key UTSW
Clinical Investigators
Clinical Trial
(NCT.gov)
Adult polyglucosan body disease Berge Minassian, M.D. Berge Minassian, M.D.  
Alzheimer's Disease Rachel Bailey, Ph.D. TBD  
Angelman Syndrome Ryan Butler, Ph.D.
Steven Gray, Ph.D.
   
Aspartyglucosaminuria (AGU) Steven Gray, Ph.D.
Xin Chen, M.D., Ph.D.
   
Charcot-Marie Tooth Type 4A Xin Chen, M.D., Ph.D. TBD  
Charcot-Marie Tooth Type 4J Steven Gray, Ph.D. Susan Iannaccone, M.D.  
CLN1 Batten Disease Steven Gray, Ph.D. Susan Iannaccone, M.D.  
CLN5 Batten Disease Steven Gray, Ph.D. Saima Kayani, M.D. NCT05228145
(Open at Univ. of Rochester)
CLN7 Batten Disease Steven Gray, Ph.D.
Xin Chen, M.D., Ph.D.
Saima Kayani, M.D.
Benjamin Greenberg, M.D.
NCT04737460
(Open at UTSW)
DDX3X Syndrome Xin Chen, M.D., Ph.D.    
ECHS1 Deficiency Rachel Bailey, Ph.D. Saima Kayani, M.D.  
FRRS1L epileptic encephalopathy Berge Minassian, M.D. Berge Minassian, M.D.  
Giant Axonal Neuropathy Steven Gray, Ph.D.
Rachel Bailey, Ph.D.
Susan Iannaccone, M.D. NCT02362438
(Open at NIH)
GNAO1 encephalopathy (GNNAO1) Xin Chen, M.D., Ph.D. TBD  
KCTD7 progressive myoclonus epilepsy Berge Minassian, M.D. Berge Minassian, M.D.  
Krabbe disease Steven Gray, Ph.D.    
La Fora disease Berge Minassian, M.D. Berge Minassian, M.D.  
Leigh Syndrome Steven Gray, Ph.D. Saima Kayani, M.D.  
Multiple Sulfatase Deficiency Rachel Bailey, Ph.D.
Steven Gray, Ph.D.
   
Non-Ketonic Hyperglycinemia (NKH) Steven Gray, Ph.D. TBD  
Peroxisome Biogenesis Disorders Ryan Butler, Ph.D. Saima Kayani, M.D.  
Pompe disease Berge Minassian, M.D. Berge Minassian, M.D.  
Prader Willi Syndrome Ryan Butler, Ph.D. TBD  
Progressive Supranuclear Palsy Rachel Bailey, Ph.D. TBD  
Riboflavin Transporter Deficiency Neuronopathy (SLC52A2) Steven Gray, Ph.D.
Xin Chen, M.D., Ph.D.
TBD  
Sandhoff Disease Steven Gray, Ph.D. Susan Iannaccone, M.D. NCT04798235
(Open at Queen’s Univ, Canada)
SLC13A5 Deficiency Disorder Rachel Bailey, Ph.D.    
SLC6A1- Related Disorder Steven Gray, Ph.D.    
Spastic Paraplegia 50 (SPG50) Steven Gray, Ph.D.
Xin Chen, M.D., Ph.D.
Susan Iannaccone, M.D.  
Steroid 5α-Reductase 3 Deficiency (SRD5A3) Steven Gray, Ph.D.
Xin Chen, M.D., Ph.D.
   
Tauopathies Rachel Bailey, Ph.D. TBD  
Tay-Sachs Steven Gray, Ph.D. Susan Iannaccone, M.D. NCT04798235
(Open at Queen’s Univ, Canada)
Unverricht-Lundborg disease Berge Minassian, M.D. Berge Minassian, M.D.  

External Partners/Collaborators/Sponsors

  • Batten Hope
  • Boston Children’s Hospital
  • Chelsea’s Hope
  • CMT Association
  • Cure ECHS1
  • Cure RTD
  • CureCMT4J
  • CureMito (CureSURF1)
  • CurePSP
  • CureSPG50
  • DDX3X Foundation
  • Drake Rayden Research Foundation
  • Finding Hope for FRRS1L
  • Global Foundation for Peroxisomal Disorders
  • Hannah’s Hope Fund
  • Ionis Pharmaceuticals
  • Jackson Laboratories
  • KCTD7 Foundation
  • Lincoln University, New Zealand
  • Mila’s Miracle
  • National Institutes of Health
  • Nationwide Children’s Hospital
  • Queen’s University, CA
  • Rare Trait Hope Fund
  • Sappani Foundation
  • SLC6A1 Connect
  • Taylor’s Tale
  • Taysha Gene Therapies
  • Tess Foundation
  • United MSD Foundation
  • University of North Carolina Chapel Hill
  • University of Pennsylvania