Area of research
Genetics · Molecular Biology
Research interest
Research interests include Virus-based gene therapy research, CAR-T cell therapy research, CRISPR and Genetic Engineering, and RNA Interference and Gene Delivery.
Lentiviral Gene Therapy for Severe Leukocyte Adhesion Deficiency Type 1
Long-Term Safety and Efficacy of Gene Therapy for Adenosine Deaminase Deficiency
Prime Editing for p47 <sup>phox</sup> -Deficient Chronic Granulomatous Disease
Hematopoietic cell transplantation for Wiskott-Aldrich syndrome: a PIDTC report
Outcomes following matched sibling donor transplantation for severe combined immunodeficiency: a report from the PIDTC
Generation of allogeneic CAR-NKT cells from hematopoietic stem and progenitor cells using a clinically guided culture method
Enhancing pediatric access to cell and gene therapies
Antiviral cellular therapy for enhancing T-cell reconstitution before or after hematopoietic stem cell transplantation (ACES): a two-arm, open label phase II interventional trial of pediatric patients with risk factor assessment
Successes and challenges in clinical gene therapy
Measuring the effect of newborn screening on survival after haematopoietic cell transplantation for severe combined immunodeficiency: a 36-year longitudinal study from the Primary Immune Deficiency Treatment Consortium
Human T cell generation is restored in CD3δ severe combined immunodeficiency through adenine base editing
Secondary Neoplasms After Hematopoietic Cell Transplant for Sickle Cell Disease
Genotype, oxidase status, and preceding infection or autoinflammation do not affect allogeneic HCT outcomes for CGD
Gene Therapy for β-Hemoglobinopathies: From Discovery to Clinical Trials
Updated Management Guidelines for Adenosine Deaminase Deficiency
Intestinal microbiome and metabolome signatures in patients with chronic granulomatous disease
Posttransplantation late complications increase over time for patients with SCID: A Primary Immune Deficiency Treatment Consortium (PIDTC) landmark study
The diagnosis of severe combined immunodeficiency (SCID): The Primary Immune Deficiency Treatment Consortium (PIDTC) 2022 Definitions
High-level correction of the sickle mutation is amplified in vivo during erythroid differentiation
Outcomes following treatment for ADA-deficient severe combined immunodeficiency: a report from the PIDTC
The diagnosis of severe combined immunodeficiency: Implementation of the PIDTC 2022 Definitions
Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency
Antiviral drug screen identifies DNA-damage response inhibitor as potent blocker of SARS-CoV-2 replication
Development of allogeneic HSC-engineered iNKT cells for off-the-shelf cancer immunotherapy
Gene Therapies for Primary Immune Deficiencies
Selected Abstracts from the 12th Annual Meeting of the Clinical Immunology Society: 2021 Virtual Annual Meeting: Immune Deficiency and Dysregulation North American Conference
Evidence generation and reproducibility in cell and gene therapy research: A call to action
Lentiviral gene therapy for X-linked chronic granulomatous disease
Excellent outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome: a PIDTC report
Artificial thymic organoids represent a reliable tool to study T-cell differentiation in patients with severe T-cell lymphopenia