Washington University in St Louis · NeuroscienceFindlay Gene Therapy Lab
Andrew Findlay · Saint Louis, Missouri
Research Focus
Dr. Andrew Findlay's lab at Washington University focuses on developing new gene therapies for muscular dystrophies, particularly a type called limb-girdle muscular dystrophy D1 (LGMDD1). Unlike other gene therapy approaches that address recessive disorders, his research targets dominantly inherited conditions where traditional methods fail. His team is pioneering a technique called allele specific knockdown (ASKD) to selectively silence harmful gene mutations while preserving normal gene function. This work is crucial for advancing the treatment landscape for similar muscular dystrophies and potentially other genetic disorders.
gene therapymuscular dystrophyallele specific knockdownLGMDD1RNA interferencetoxic gain-of-functionsiRNAgenetic disordersmyopathy
Research Aims
3AIM 01Develop a selective knockdown strategy for mutant DNAJB6 to improve disease phenotypes in LGMDD1 mouse models.
AIM 02Validate the efficacy of allele specific knockdown in vitro using human models of LGMDD1.
AIM 03Optimize allele specific knockdown targeting to expand therapeutic applicability for multiple DNAJB6 mutations.
Skills the Lab Values
RNA interferencemouse modelsinduced pluripotent stem cellsWestern blottingproteomicscell culturegenetic engineeringreverse transcriptase polymerase chain reaction (RT-PCR)gene correctiondata analysis using genetic databases
Output against the department
Publications53bottom 12% of dept.588th of 671
this lab
most publishedleast published
Citations1.1kbottom 6% of dept.632nd of 671
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.