Abdullah Khan

Working in the Pediatric Hematology division at Johns Hopkins School of Medicine, I focus on developing non-viral gene therapy approaches using CRISPR-based base editing and lipid nanoparticle (LNP) delivery, with a primary focus on Ataxia-Telangiectasia. I design and execute in vivo RNA base editing studies targeting pathogenic ATM variants across the central nervous system, liver, lung, and blood using optimized delivery routes including intravenous, intrathecal, and intracerebroventricular administration. On the formulation side, I develop and characterize LNPs using Zetasizer-based analysis, measuring particle size, zeta potential, and PDI to ensure delivery efficiency and stability. I apply Quality by Design principles to optimize dosing regimens and tissue-specific targeting strategies, including nanoprimer-based liver saturation approaches. At the cellular level, I work with mammalian cell culture and iPSC maintenance, perform transfections and electroporation, and carry out PCR, gel electrophoresis, DNA/RNA extraction, and NGS-based validation of editing outcomes. I use Benchling and SnapGene for gRNA design, plasmid mapping, and off-target prediction, and analyze results using GraphPad Prism and ImageJ. To assess translational feasibility, I integrate IVIS imaging, tissue processing, sequencing-based editing analysis, and toxicity monitoring into my workflow, evaluating biodistribution, editing precision, off-target effects, and functional restoration of ATM activity across treated tissues.

Formulated and characterized 100+ distinct LNP libraries using Zetasizer-based particle characterization (size, zeta potential, PDI) and DLS to optimize delivery efficiency and stability, while building in vivo expertise in LNP-mediated RNA delivery and CRISPR base editing across CNS, blood, lung, and liver tissues through tissue-specific targeting and dosing optimization. Executed a broad range of molecular and cellular biology techniques including mammalian cell culture (HEK293T, Neuro2a, Fibroblasts, AML12), iPSC maintenance and transfection, PCR, gel electrophoresis, primer design, base editing, NGS validation, DNA/RNA extraction, and fluorescence/confocal microscopy. Applied CRISPR-based base editing and bioinformatics tools (Benchling, SnapGene) for gRNA design, plasmid mapping, and off-target prediction, and conducted translational in vivo assessments integrating IVIS imaging, flow cytometry, sequencing-based editing analysis, and toxicity monitoring to advance non-viral gene therapy development. Mastered diverse in vivo procedures including eight routes of administration (Intravenous, Intraperitoneal, Per os (Oral), Intranasal, Intrathecal, Retro-orbital, Intradermal, Subcutaneous), multiple blood collection methods, and full necropsy workflows (organ collection, cardiac bleed, BAL), while strengthening quantitative analysis skills through GraphPad Prism, ImageJ, and collaborative precision medicine research.

Applied expertise in sterility testing and rapid microbiological methods (RMM) by validating PCR-based and ATP bioluminescence assays, reducing detection time by 40% and improving efficiency by 30%, while utilizing automated and manual colony counting to boost testing accuracy by 25% and streamline workflows across 10+ laboratories, cutting processing time by 15%. Trained personnel in aseptic handling and GMP-compliant microbial testing, ensuring regulatory compliance across 20+ client audits, and delivered product demonstrations, technical support, and SOP/training material development for sterility testers and differential counters.

Characterized 50+ microbial communities using flow cytometry and mass spectrometry; isolated and cultured diverse microorganisms with advanced media formulations.

Performed quality control analyses (HPLC, UV-Vis) per cGMP/GLP guidelines, achieving 100% batch release compliance for pharmaceutical stability studies.

GPA: 3.9/4.0

GPA: 3.9/4.0

Student Council Member, Active Debater, Table Tennis Captain, Inquisitive Club President