Philadelphia, Pennsylvania, United States
Translational molecular biologist with in vitro/in vivo expertise in preclinical development of cell and gene therapies for metabolic and neurological disorders, seeking opportunities to continue developing novel therapeutics. Experience includes (but is not limited to) CRISPR-based gene editing (base and prime editing), neurological and neurodegenerative diseases, rare diseases, inborn errors of metabolism, ex vivo lentiviral gene therapy, hematopoietic stem cell transplant, and iPSC-derived models of disease.
Laboratories of Kiran Musunuru, MD, PhD, MPH, ML, MRA (University of Pennsylvania) and Rebecca Ahrens-Nicklas, MD, PhD (Children's Hospital of Philadelphia) • Design and execute CRISPR-based genome engineering strategies (including base and prime editing) for targeted correction of inherited metabolic disorders. • Develop and optimize guide RNAs and targeted integration approaches; validate editing outcomes using NGS, qRT-PCR, and ddPCR. • Generate lentiviral-engineered cell models to evaluate gene delivery strategies and editing efficiency in a platform-based approach. • Perform molecular cloning and construct design for precise genome modification workflows. • Collaborate with industry partners to assess novel gene delivery technologies. • Maintain rigorous experimental documentation and present findings to multidisciplinary scientific and translational teams.
University of Pennsylvania, Perelman School of Medicine Cell and Molecular Biology Graduate Group | Gene Therapy and Vaccines Program Laboratory of Rebecca Ahrens-Nicklas, MD, PhD (Children's Hospital of Philadelphia) • Performed and optimized the preclinical development of ex vivo lentiviral gene therapy with hematopoietic stem cell transplant for multiple sulfatase deficiency and related neuronopathic lysosomal storage disorders • Generated and characterized novel patient iPSC-derived neuron and microglia cell models of multiple sulfatase deficiency to dissect disease pathophysiology and examine cross-correction mechanisms after gene therapy • Determined translational phenotypic endpoints for therapeutic rescue at the molecular, cellular, and behavioral (including neurological and motor assays) levels through extensive work with both in vitro and in vivo models of disease
Laboratory of Jonathan Raper, PhD (Department of Neuroscience) • Determined the molecular mechanisms of olfactory sensory neuron axon guidance in the developing olfactory bulb of larval zebrafish utilizing novel transgenic fluorescent lines, CRISPR/Cas9 editing, and confocal microscopy
Biogen CoLab • Designed and performed cell and molecular biology experiments with high school students from marginalized communities in order to cultivate their interests in biomedical research • Gained expertise in lab techniques such as E. coli microbial culture, bacterial transformation, plasmid mini-prep, site-directed mutagenesis, SDS PAGE, and purification by batch affinity chromatography • Led group classes (20-40 students) to review experimental ideology and techniques and apply concepts in a hands-on laboratory setting
• Assessed metacognitive awareness in higher-functioning children and adolescents with Autism Spectrum Disorder (ASD) • Communicated with and interviewed community members to elicit participation in the study, conduct assessments, and follow up with participants • Created advertisements and designed recruitment techniques to draw in study participation
• Aided senior thesis students by running the electroencephalogram (EEG) on human subjects in order to test the effectiveness of reappraisal techniques on emotion recognition • Collaborated with fellow student researchers and academic advisors to collect and analyze data