UMCUniversity of Massachusetts Chan Medical School · Genetics
Gene Therapy Innovations Lab
Heather L Gray-Edwards · Worcester, Massachusetts
Research Focus
Dr. Heather L Gray-Edwards leads research focusing on innovative gene therapies for severe genetic disorders such as Maple Syrup Urine Disease (MSUD) and Sialidosis. Her lab utilizes animal models, including mice and sheep, to test adeno-associated virus (AAV) gene therapies that aim to correct underlying biochemical issues and alleviate associated neurological symptoms. The overarching goal is to develop effective treatments that can prevent the severe complications often seen in these disorders, ultimately paving the way for future clinical applications in human medicine.
gene therapyMaple Syrup Urine DiseaseSialidosisAAV vectorneurological disordersanimal modelsCRISPRtherapeutic developmentclinical trials
Research Aims
3AIM 01Evaluate the efficacy of high-dose AAV gene therapy to treat neurological issues in MSUD.
AIM 02Conduct long-term assessments of gene therapy strategies in large animal models.
AIM 03Develop a sheep model of Sialidosis to refine treatment approaches before human trials.
Skills the Lab Values
Adeno-associated virus (AAV) gene therapymouse modelssheep modelsCRISPR/Cas9 gene editingclinical trial designtoxicology studiestranscriptomicsmetabolomicsMRI imagingdose-response studies
Output against the department
Publications90bottom 27% of dept.188th of 259
this lab
most publishedleast published
Citations2.2kbottom 8% of dept.237th 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.