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Office
1201 W La Veta Blvd
Orange, CA 92868Phone+1 714-509-8852Fax+1 714-509-4531
Summary
- I am the director of multidisciplinary lysosomal storage disorder program at CHOC Children’s and a board certified clinical geneticist and biochemical genetics specialist.
Education & Training
- Cedars-Sinai Medical CenterFellowship, Medical Genetics and Genomics, 2006 - 2007
- Cedars-Sinai Medical CenterResidency, Pediatrics, 2001 - 2006
- David Geffen School of Medicine at UCLAClass of 2001
Certifications & Licensure
- CA State Medical License 2002 - 2026
- American Board of Medical Genetics and Genomics Clinical Biochemical Genetics
- American Board of Medical Genetics and Genomics Clinical Genetics and Genomics
Clinical Trials
- RGX-111 Gene Therapy in Patients With MPS I Start of enrollment: 2019 Apr 03
- Longitudinal Assessment of Atypical Tripeptidyl Peptidase 1 Enzyme Deficiency Patients Start of enrollment: 2019 Nov 01
- A Safety and Efficacy Study of LYS-GM101 Gene Therapy in Patients With GM1 Gangliosidosis Start of enrollment: 2021 May 11
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Publications & Presentations
PubMed
- 17 citationsApplication of N-palmitoyl-O-phosphocholineserine for diagnosis and assessment of response to treatment in Niemann-Pick type C diseaseRohini Sidhu, Pamela Kell, Dennis J. Dietzen, Nicole Y. Farhat, An Ngoc Dang Do
Molecular Genetics and Metabolism. 2020-01-22 - 11 citationsApplication of a glycinated bile acid biomarker for diagnosis and assessment of response to treatment in Niemann-pick disease type C1.Rohini Sidhu, Pamela Kell, Dennis J. Dietzen, Nicole Y. Farhat, An Ngoc Dang Do
Molecular Genetics and Metabolism. 2020-11-18 - 46 citationsBiochemical, molecular, and clinical characteristics of children with short chain acyl-CoA dehydrogenase deficiency detected by newborn screening in CaliforniaNatalie M. Gallant, Karen Leydiker, Hao Tang, Lisa Feuchtbaum, Fred Lorey
Molecular Genetics and Metabolism. 2012-05-01
Press Mentions
- Gene Therapy in Children with MPS I Showing PromiseMarch 8th, 2023
- UCI Earns Designation as CIRM Alpha Clinic with Five-Year, $8 Million GrantOctober 27th, 2022
- Lysogene Provides Additional Update on AAVance Phase 2/3 Gene Therapy Clinical Trial with LYS-SAF302 in Children with MPS IIIAJuly 7th, 2022
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Grant Support
- Combination Gene Therapy for Treatment of Canine Mucopolysaccharidosis Type IEunice Kennedy Shriver National Institute of Child Health and Human Development2023–2028
- Alpha Stem Cell ClinicCalifornia Institute of Regenerative Medicine2023–2028
- Stable therapy in Pompe disease through genome editingNational Institute of Arthritis and Musculoskeletal and Skin Disease2021–2026
Committees
- Member, CalOptima Pharmacy and Therapeutics Committee 2018 - Present
- Member, CHOC Clinical Efficacy and Efficiency Committee 2018 - Present
Research History
- Principal InvestigatorAs PI of the Campbell Foundation of Caring Laboratory, I supervise efforts to develop CRISPR-based gene editing methods for generation of model systems and therapies for inherited lysosomal storage disorders.2017 - Present
Professional Memberships
- Member
- Society of Inherited Metabolic DiseaseMember
- Society for the Study of Inborn Errors of MetabolismMember
- American Society of Cell and Gene TherapyMember
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