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. |
Kim Goodspeed, M.D. |
|
| Aspartyglucosaminuria (AGU) |
Steven Gray, Ph.D.
Xin Chen, M.D., Ph.D. |
Kim Goodspeed, M.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. |
Kim Goodspeed, M.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. |
Kim Goodspeed, M.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. |
Kim Goodspeed, M.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. |
Kim Goodspeed, M.D. |
|
| SLC6A1- Related Disorder |
Steven Gray, Ph.D. |
Kim Goodspeed, M.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. |
Kim Goodspeed, M.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
