Zhonglin Z.

Experienced research scientist specializing in the biological mechanisms underlying lysosomal storage disorders (Fabry, Niemann-Pick type C, MLD, Pompe disease) and X-linked adrenoleukodystrophy (X-ALD), as well as disorders involving Emopamil-binding protein (EBP) and Galactosemia. Proven track record in leading cross-functional research programs from early discovery through late-stage validation. Key Contributions Project Lead – Galactosemia Program Led multidisciplinary teams across DMPK, formulation, pathology, analytical R&D, chemistry, and translational medicine, as well as external collaborators. Oversaw initial assay development and target validation efforts. Biology Lead – X-linked Adrenoleukodystrophy (X-ALD) Initiated and directed both in vitro and in vivo target validation, advancing the project to late-stage development. Managed PPMO in vivo target validation for X-ALD and Galactosemia programs. EBP Project Conducted target credentialing studies in disease fibroblasts and rat oligodendrocyte precursor cells (OPCs). Genotype–Phenotype Correlation Studies Investigated genotype–phenotype relationships using CRISPR whole-genome library screening to identify disease-relevant targets and pathways.

During this period, I have solved many technical challenges for different disease indications, including refining animal models and setting up many in vitro assays for various projects. 1. Designed & characterized genetically (AAV) modified animal model to study lipid movement in Fabry disease. 2. Developed highly accurate HPLC plasma fractionation method to study lipid distribution 3. Implemented siRNA, ZFN/TALEN/Crispr, and Lenti shRNA K.D. Methods for in vitro POC, generating stable cell lines by using Crispr 4. Developed and optimized IHC and biochemical cholesterol detection assays 5. Studied and characterized biomarker and transcription regulation changes in diabetes and NPC animal, models

Scientist, Dept. of Pharmacology and Preclinical Research Explored mineral metabolism in chronic kidney disease (CKD) setting, with a focus on intestinal phosphate absorption, bone phosphate and calcium storage, and kidney secretion 1. Optimized chemically induced uremia model for the study of phosphate transport, advanced glycation end products (AGE), and vascular calcification. 2. Designed and carried out due diligence efforts on a bone morphogenetic protein (BMP) antagonist. 3. Developed highly sensitive platelet aggregation and whole blood assays for therapeutic polymer projects. 4. Develop direct and indirect ELISAs for AGE, bone markers, renal, inflammatory, and polymer projects. 5. Provided technical support in a QA-GLP setting to detect viral contaminants in Genzyme's bioreactors. 6. Performed MTD in vivo experiments to support new therapeutic approaches. 7. Extensively tested lead compounds in PK/PD and efficacy models, especially for Napi-2b inhibitor.

Advanced knowledge and extensive experience with HSC, including culture, expansion, transfection, formulation, and standard immunological assays such as proliferation, cytokine FACS, and ELISAs, and multiplex assays, etc. in the research areas of inflammation, cancer, and autoimmune disease Calcium Release - Activated Calcium (CRAC) Project • Established and conducted OZ-induced colitis (IBD), Allergic Contact Dermatitis (ACD), Delayed-Type Hypersensitivity (DTH), and Experimental Autoimmune Encephalomyelitis (EAE) in vivo models • Optimized rat Collagen-Induced Arthritis (CIA) and Adjuvant-Induced Arthritis (AIA) animal models • Performed animal models of DNBS-induced colitis (IBD) and collagen-Induced Arthritis (CIA). • Detected cytokine levels in tissues and plasma using ELISA (Bioplex system and regular ELISA) • Quantified inflammation using myeloperoxidase (MPO) and C-reactive Protein (CRP) assay • Designed and performed in vivo TH1 and TH2 mouse models • Immunized TH1 model with TBC (H37Ra) in IFA and neutralized with IL-4 antibody Immunized TH2 model with Ascaris adjuvant and neutralized TH2 response with IFNγ. Vascular Disrupting Agent (VDA) Inhibitor Project 1. Designed IC50 assay for Vascular disrupting agents (VDA) inhibitors, leader compound selected for IND 2. Designed visualization assay for microtubule polymerization and depolymerization 3. Selected compounds with anti-vascular activity by HUVEC formation and disruption assay

1. Designed, performed, and analyzed real-time PCR to identify gene expression in the mouse brain cortex. 2. Validated differential cell expression in mouse brain cortex by in situ hybridization 3. Experienced in Biocytin Staining, small animal tattoo, and mouse genotype technique

Center for Engineering in Medicine, Shriners Hospital for Children, Boston, MA 1. Optimized polydimethylsiloxane (PDMS), collagen, and fibronectin-coated surfaces for uniform cell culture 2. Designed micro-patterned surfaces using a mechanical tool (AutoCAD) to promote cell survival to develop bioactive bandages. 3. Performed viability and functional assays for desiccated human 3T3 cells

Masters Thesis: Development of polylaminate Fabrics based bioreactors