Maja Haerle

Mentor:

Andrew Berglund 

College of Medicine

Major: Biochemistry 

Minor: N/A

Research Interests: Genetics, Biophysics, Biochemistry

Academic Awards: Johns Hopkins All Children’s Hospital Frances Shoemaker Scholarship 2016, Bay News 9 Project Weather Scholarship 2016, Emerging Scholars Program 2016-2017.

Organizations: UF Medical Journal Society, UF Undergraduate American Medical Women's Association (UAMWA), 

Volunteer: UF Health Shands Hospital, UF Children Beyond Our Borders (CBOB) College Prep Mentoring Program, 

Hobbies and Interests: Cooking/baking, Running, Watching soccer

Research Description:

Engineering Zinc Finger Proteins to Target CTG DNA Repeat Expansions in Myotonic Dystrophy Type 1 (DM1)

Myotonic dystrophy (DM) is a genetic disorder and the most common form of adult muscular dystrophy. Myotonic dystrophy type 1 (DM1) is caused by tri-nucleotide expansions (CTG) in DNA which lead to production and accumulation of expanded toxic CUG non-coding RNAs after the transcription process. As a result, two RNA-binding proteins MBNL 1 (or muscleblind-like protein) and CUGBP 1 (or CUG-binding protein 1) cause a disruption and deregulation of splicing and mRNA translation, stability, and function.
Through the design and implementation of DNA binding proteins, I hope to achieve a transcription "blockade" which can prevent the harmful and toxic effects of the RNA generated from the microsatellite expansions. The zinc-finger (ZF) protein is one of the most common DNA binding molecules and is composed of two beta sheets, an alpha helix, and a zinc atom coordinated by cysteine and histidine residues. I will be focused on using ZF proteins to repress the transcription of genes and will take part in making designer DNA binding factors (proteins that bind DNA and regulate transcription through their DNA-binding domains (ZF-DBDs) and effector domains). The engineered ZF-DBDs will be cloned, purified, and directly delivered to mammalian cells.