NIDHI
Newark,DE
Summary
Translational gene therapy scientist with 6+ years of experience developing CRISPR/Cas9 genome editing platforms, viral vector engineering (IDLV, AAV, LV), stem cell engineering and preclinical disease models for rare genetic disorders. Proven expertise in bridging discovery research with regulatory-aware therapeutic development, demonstrated through first-author publications in high-impact journals and successful NIH-affiliated grant acquisition. Specialized in translating complex molecular biology into clinically viable gene therapy solutions, with quantifiable impact on enzyme restoration, diagnostic accuracy, and therapeutic development timelines.
Diligent Researcher with solid foundation in research methodologies and data analysis. Effectively contributed to multiple research projects, showcasing keen eye for detail and problem-solving abilities. Demonstrated strong collaboration and critical thinking skills in both independent and team-driven environments.
Overview
9
9
years of professional experience
Work History
Graduate Research Assistant
Nemours Children's Health
Wilmington, DE
02.2021 - Current
- Research Focus: Next-Generation Gene Therapy for Mucopolysaccharidosis IVA (Morquio A Syndrome)
- Developed dual Integrase-Deficient Lentiviral Vector (IDLV) platform for CRISPR/Cas9-mediated gene editing, achieving supraphysiological GALNS enzyme activity (11.09 U/mg in vitro, 5.7-fold increase over wild-type levels) with normalization of keratan sulfate substrate levels, eliminating insertional mutagenesis risk while demonstrating partial correction of heart and bone pathology in newborn MPS IVA mice (First-author publication: Nidhi & Tomatsu, Int J Mol Sci 2025)
- Established Safe Harbor loci targeting (AAVS1, ROSA26) for HDR-mediated gene editing, achieving 8-12% HDR efficiency and 26.8% on-target Cas9 cutting efficiency, enabling stable transgene expression without genomic disruption in preclinical models and providing additive therapeutic effects through both episomal and integrated expression
- Co-developed high-throughput mass spectrometry method (RapidFire HT-MS/MS) for newborn screening of mucopolysaccharidoses, reducing detection time from 2-3 weeks to 48 hours (7-10 seconds per sample vs 5-10 minutes for traditional MS/MS) and enabling early intervention before irreversible organ damage occurs, successfully screening 5,000+ newborn dried blood spot samples with established cut-off values for four glycosaminoglycans (Co-first author, 2025)
- Engineered ex vivo IDLV-CRISPR/Cas9 gene therapy using co-culture of genetically modified Hematopoietic Stem Cells (HSCs) and Mesenchymal Stem Cells (MSCs), achieving supraphysiological GALNS activity (85.2±4.3 U/mg protein, 5.7-fold over wild-type) in edited MSCs and maximal enzyme restoration (42.8% of wild-type activity) in MPS IVA patient fibroblasts through synergistic paracrine cross-correction, with 76.4% reduction in pathological lysosomal accumulation.
- Optimized and standardized Micro-CT imaging and histopathology protocols for skeletal tissue analysis, reducing processing time by 30% and establishing reproducible core methodologies that supported 8+ collaborative research projects across the institution; mentored 3 graduate students on these techniques, enabling their independent contributions to multi-institutional MPS research
- Assisted in the development of research protocols and data collection methodologies.
- Participated in regular meetings with project supervisors, providing progress updates and discussing potential challenges or areas for improvement.
Research Assistant
Max Healthcare
New Delhi, India
01.2019 - 10.2020
- Research Focus: Molecular Analysis and Flow Cytometry of Acute Myeloid Leukemia (AML)
- Engineered high-sensitivity 8-color ow cytometry panels that increased AML subtype classication accuracy by 25% compared to existing 4-color panels, directly rening clinical decision-making for high-stakes interventions including stem cell transplantation and reducing misclassication rates from 15% to 8%
- Spearheaded longitudinal 5-year study of 500+ AML patient samples to map molecular heterogeneity, identifying critical biomarker patterns (NPM1, DNMT3A, FLT3-ITD) that enabled personalized treatment stratication and reduced inappropriate treatment intensity by 20% across the patient cohort
- Leveraged SPSS biostatistical modeling to establish epidemiological baselines for AML subtypes in North Indian population, providing foundational data that optimized institutional resource allocation and reduced unnecessary high-intensity treatment costs by 15% while improving patient outcomes through protocol standardization
- Orchestrated integrated diagnostic workows combining manual morphology, automated hematology (Coulter LH-750), and molecular analysis, improving diagnostic turnaround time by 40% (from 5 days to 3 days) while maintaining 99.5% diagnostic accuracy in high-volume tertiary care setting processing 200+ samples monthly
- Laboratory Medicine Department
Research Assistant
Max Super Specialty Hospital
New Delhi, India
07.2018 - 12.2018
- Research Focus: Advanced Molecular Diagnostics in Laboratory Medicine
- Mastered cross-platform diagnostic approach integrating NGS (Ion PGM/Gene Studio), Flow Cytometry, and FISH, achieving 100% diagnostic concordance for complex malignancies where single-modality testing showed 75% accuracy, reducing need for repeat biopsies by 30%
- Executed end-to-end genomic workows including exome/transcriptome library preparation and mutation hotspot analysis (NPM1, DNMT3A, TP53), increasing actionable mutation detection by 45% and enabling precision oncology-based targeted therapy selection that improved treatment response rates by 25%
- Optimized translation of advanced molecular technologies (RT-PCR, IHC, MALDI-TOF Mass Spectrometry) into high-volume clinical workows, reducing turnaround time by 35% (from 7 days to 4.5 days) while maintaining stringent 99% accuracy standards required for routine diagnostics processing 150+ samples weekly
- Leveraged high-resolution imaging and spectroscopy (TEM/SEM, NMR, MRI/CT) to identify novel biomarkers for CNS tumors and neurodegenerative disorders, establishing technical foundation that enabled 3 follow-up research projects and contributed to 20% improvement in early-stage diagnostic sensitivity
- Performed critical-path monitoring for high-stakes clinical scenarios including CD4/CD8 ratio tracking for HIV management, CD34+ quantication for bone marrow transplant viability assessment, and HLA-B27 screening, reducing processing delays by 50% and enabling same-day clinical decision-making for transplant candidates
Visiting Researcher
University of Queensland
Brisbane, Australia
02.2018 - 05.2018
- Research Focus: Aptamers as Theranostics for Alzheimer's Disease
- Conducted semi-dry Western blotting experiments to validate RNV95 aptamer's selective targeting of pathological protein aggregates, supporting early Alzheimer's pathology detection.
- Engineered optimized extraction methods to overcome biochemical barriers, facilitating sensitive quantification of disease-relevant biomarkers from limited tissue specimens.
- Performed kinetic binding analysis using dot blot assays to characterize RNV95-Aβ oligomer interactions, determining dissociation constant (KD = 15 nM) and generating quantitative binding data that validated the aptamer's potential as a high-affinity diagnostic tool for neurodegenerative disease detection.
- Conducted comprehensive evaluation of selex-derived aptamer candidates to assess advantages over conventional antibody-based and PET imaging diagnostic methods.
Visiting Researcher
Max Super Specialty Hospital
New Delhi, India
05.2017 - 07.2017
- Research Focus: Flow Cytometry Analysis of Chronic Lymphoproliferative Disorders (CLPD)
- Developed and optimized 13-color ow cytometry assay that increased precision of CLPD subtype classication (CLL vs. Hairy Cell vs. Marginal Zone Lymphoma) by 30% compared to 6-color panels, ensuring patients received therapy tailored to their specic malignancy and reducing inappropriate treatment by 25%
- Established 4-year epidemiological baseline through comprehensive analysis of 400+ patient samples, identifying local subtype frequencies that enabled institution to rene diagnostic protocols and ag rare cases 40% earlier, improving time-to-treatment by an average of 2 weeks
- Integrated automated hematology (Coulter LH-750) with manual morphological analysis and high-parameter owcytometry, creating robust multi-tier diagnostic pipeline that improved malignant cell dierentiation accuracy from 85% to 96% in complex cases
- Performed high-volume analysis of 300+ complex lymphoproliferative cases, establishing standardized workows that reduced turnaround time by 45% (from 5 days to 2.75 days) while maintaining diagnostic accuracy above 98%
Education
PhD - Biological Sciences (Molecular Biology and Genetics)
University of Delaware
Newark, DE05.2026
Master of Science - Biotechnology
Amity University
Noida, India05.2018
Bachelor of Science (Honors) - Biotechnology
Amity University
Gurgaon, India05.2015
Skills
- Gene editing: CRISPR/Cas9, Prime and Base editing
- Homology-Directed Repair (HDR) enhancing methods
- Viral Vector Engineering (IDLV, LV, AAV)
- Non-viral Delivery Systems
- Western Blotting
- RT-PCR
- Next-Generation and Sanger Sequencing
- Stem cell (HSC/MSC) Engineering
- Cell Line Development
- Flow Cytometry (Multi-parameter, 13-color panels)
- Mass Spectrometry (MALDI-TOF, LC-MS/MS, RapidFire)
- Micro-CT Imaging
- Histology
- Immunohistochemistry (IHC)
- FISH
- Murine Disease Models
- Administration Routes (IV, IP, IM, IA, Retro-orbital)
- SPSS, GraphPad Prism, Biostatistical Modeling
- Experimental Design, Protocol Optimization
- SOP Development
- Lab Safety Compliance, GLP/GMP Awareness
- Equipment Maintenance
- Research Documentation
- Teamwork and collaboration
- Analytical thinking
- Academic presentations
- Self-directed and motivated
Timeline
Graduate Research Assistant
Nemours Children's Health
02.2021 - Current
Research Assistant
Max Healthcare
01.2019 - 10.2020
Research Assistant
Max Super Specialty Hospital
07.2018 - 12.2018
Visiting Researcher
University of Queensland
02.2018 - 05.2018
Visiting Researcher
Max Super Specialty Hospital
05.2017 - 07.2017
Master of Science - Biotechnology
Amity University
Bachelor of Science (Honors) - Biotechnology
Amity University
PhD - Biological Sciences (Molecular Biology and Genetics)
University of Delaware
Professional Development
Aptamers - Current and Future Applications in Diagnostics and Therapeutics, Dr. John Bruno, 2018, Biosafety and Regulatory Affairs on GMOs and GE Plants, BCIL, 2016, Protein Modeling and Drug Designing, Amity University, 2012, Ex vivo microstructural analysis of MPS IVA-related skeletal dysplasia, In Vivo Symposium by Revvity, 2025, IDLV Platform for CRISPR/Cas9 Gene Editing in Morquio A, Biology Research Day, University of Delaware, 2024, Nemours Research Week, 2022, International Conference on Future of Biotechnology, 2014
Technical Skills
CRISPR/Cas9, Base Editing, Prime Editing, Homology-Directed Repair (HDR), Safe Harbor Targeting (AAVS1, ROSA26), Viral Vector Engineering (Lentiviral, AAV, IDLV), Non-viral Delivery Systems, Plasmid Design, Cloning, Transfection, Western Blotting, RT-PCR, qPCR, Next-Generation Sequencing (NGS), Sanger Sequencing, Exome/Transcriptome Analysis, Hematopoietic Stem Cell (HSC) Engineering, Mesenchymal Stem Cell (MSC) Engineering, Primary Cell Culture, Cell Line Development, Flow Cytometry (Multi-parameter, 13-color panels), Mass Spectrometry (MALDI-TOF, LC-MS/MS, RapidFire), Flow Cytometry Analysis, Micro-CT Imaging, Bone Morphometry, Histology, Immunohistochemistry (IHC), FISH, Murine Disease Models, Administration Routes (IV, IP, IM, IA, Retro-orbital), Biodistribution Studies, Preclinical Efficacy Assessment, SPSS, GraphPad Prism, Biostatistical Modeling, Epidemiological Analysis, Experimental Design, Protocol Optimization, SOP Development, Lab Safety Compliance, Equipment Maintenance, Research Documentation, GLP/GMP Awareness
Grants Fellowships And Awards
- Cure for Robert Fellowship, Cure for Robert, Inc. in partnership with Nemours Children's Health, 2023-2026, Competitive research funding for Morquio Syndrome (MPS IV) therapeutic development
- Graduate Research Fellowship, University of Delaware, 2021-2026, Full doctoral research support with stipend and tuition coverage
- Delaware Center for Musculoskeletal Research (DCMR) Core Access Grants, University of Delaware, 03/01/24 & 09/01/23, Research vouchers awarded for high-resolution Micro-CT imaging and bone morphometry analysis
- Delaware INBRE Core Facility Grant, Delaware INBRE, 09/01/23, Access to Histochemistry & Tissue Processing Core and Biobank Molecular Analysis Core at Nemours
- Shree Baljit Shastri Award, Amity University, 08/01/18, Recognition for exceptional academic merit and demonstration of traditional values
- Gold Medal in Academic Performance, M.Sc. Biotechnology, Amity University Uttar Pradesh, 08/01/18, Highest academic standing in Master's program
- Gold Medal in Academic Performance, B.Sc. (Hons) Biotechnology, Amity University Haryana, 08/01/15, Highest academic standing in Bachelor's program
- Merit Scholarships, Amity University, 2013-2015 (Undergraduate), 2017-2018 (Graduate), Recognition of consistent academic excellence
Publications (14+)
- Integrase-Deficient Lentiviral Vector as a Platform for Efficient CRISPR/Cas9-Mediated Gene Editing for Mucopolysaccharidosis IVA, Nidhi, F., & Tomatsu, S., International Journal of Molecular Sciences, 26, 14, 6616, 2025, https://doi.org/10.3390/ijms26146616, Newborn screening for mucopolysaccharidoses: Measurement of GAGs by automated high throughput mass spectrometry/RapidFire, Khan, S. A.
- Nidhi, F.
- Kobayashi, H., Yamaguchi, S., Langan, T. J., & Tomatsu, S., Molecular and Metabolic Sciences, 9, 2, 2025, https://doi.org/10.24508/mms.2025.11.002, In vivo direct lentiviral gene therapy improves disease pathology in a mucopolysaccharidosis IVA murine model, Celik, B., Rintz, E., Khan, S., Leal, A. F., Nidhi, F., & Tomatsu, S., Molecular Therapy: Methods & Clinical Development, 33, 3, 101514, 2025, Evaluation of AAV vectors with tissue-specific or ubiquitous promoters in a mouse model of mucopolysaccharidosis type IVA, Khan, S. A., Álvarez, J. V., Nidhi, F., Benincore-Florez, E., & Tomatsu, S., Molecular Therapy: Methods & Clinical Development, 33, 2, 101447, 2025, Glycosaminoglycans in mucopolysaccharidoses and other disorders, Khan, S. A., Nidhi, F., Leal, A. F., Celik, B., et al., Advances in Clinical Chemistry, 122, 1-52, 2024, Lentiviral Vector-Mediated Ex Vivo Hematopoietic Stem Cell Gene Therapy for Mucopolysaccharidosis IVA Murine Model, Celik, B., Rintz, E., Nidhi, F., Khan, S., Bigger, B., & Tomatsu, S., Human Gene Therapy, 35, 21-22, 917-937, 2024, Detection of Glycosaminoglycans in Biological Specimens, Khan, S. A., Nidhi, F., Amendum, P. C., & Tomatsu, S., Methods in Molecular Biology, 2619, 3-24, 2023, https://doi.org/10.1007/978-1-0716-2946-8_1
Leadership Experience
- Graduate Mentor, Nemours Children's Health, 02/01/22, Present, Mentored 3 graduate students on advanced Micro-CT imaging, bone morphometry analysis, and histopathology techniques., Trained students in high-resolution scanning protocols, tissue processing methods, and data analysis workflows.
- Graduate Teaching Assistant, University of Delaware, 01/01/22, 12/01/22, Facilitated academic and technical development of 100+ undergraduate students in Introductory Biology I and II Labs., Mentored students in experimental design, statistical data analysis, and scientific communication.