UMCUniversity of Massachusetts Chan Medical School · Genetics
Gene Therapy and Neurological Disorders Unit
Toloo Taghian · Worcester, Massachusetts
Research Focus
Dr. Toloo Taghian's lab is focused on developing innovative gene therapy approaches for treating UBA5 deficiency, a rare genetic disorder that causes severe neurological issues in children. Using a newly created mouse model that mimics the human disease, the lab aims to understand the underlying mechanisms of UBA5 deficiency and test promising gene therapy strategies to potentially rescue neurological functions. Their research could pave the way for effective treatments for patients suffering from this deadly condition.
gene therapyUBA5 deficiencyAAV vectorneurological disordersbiomarkers
Research Aims
3AIM 01Identify and characterize the Uba5 mouse model to uncover disease mechanisms.
AIM 02Test various adeno-associated virus (AAV) vectors for effective gene delivery and treatment in mice.
AIM 03Discover biomarkers for UBA5 deficiency using metabolomic profiling.
Skills the Lab Values
mouse modelsgene delivery techniquesintracerebroventricular injectionsMRI imagingbioinformaticsmetabolomic studies
Output against the department
Publications36bottom 6% of dept.243rd of 259
this lab
most publishedleast published
Citations485bottom 2% of dept.255th of 259
this lab
most citedleast cited
Career totals from OpenAlex, compared against labs that share a department. A department spans everything from one paper to tens of thousands, so bar heights are on a log scale — otherwise the largest lab would flatten every other bar to a line.