UMCUniversity of Massachusetts Chan Medical School · Genetics
Khvorova Lab
Anastasia Khvorova · Worcester, Massachusetts
Research Focus
Dr. Anastasia Khvorova's lab at the University of Massachusetts focuses on creating new types of therapy using small pieces of genetic material called oligonucleotides. These therapies target and reduce the expression of harmful genes related to neurodegenerative diseases like Huntington's disease and ALS. The lab is working on improving the delivery of these therapies to the central nervous system to make them more effective and longer-lasting.
therapeutic oligonucleotidesneurodegenerative disordersgene silencingsiRNAdrug delivery
Research Aims
5AIM 01Develop new oligonucleotide chemistries for brain delivery.
AIM 02Characterize rapid distribution and therapeutic effects in central nervous system.
AIM 03Optimize multi-target silencing capability of oligonucleotides.
AIM 04Establish a platform for targeting any gene in the CNS.
AIM 05Create combinatorial treatments for Huntington's disease and ALS.
Skills the Lab Values
siRNA designchemical synthesisanimal modelscerebrospinal fluid infusiongene targeting
Output against the department
Publications208top 40% of dept.103rd of 259
this lab
most publishedleast published
Citations31ktop 19% of dept.48th 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.