Excellence in Research Award

About the Award

Each year the ASGCT Abstract Review Committee selects 15 top abstracts submitted by students and fellows to receive Excellence in Research Awards. Recipients of the Excellence in Research Awards are announced during the George Stamatoyannopoulos Memorial Lecture at the ASGCT Annual Meeting and receive a monetary award of $500 along with a plaque.

Criteria and Selection

Any ASGCT Member or Associate Member who submits an abstract to the Annual Meeting is eligible to receive the award.



Sangya Agarwal
Investigating the Therapeutic Efficacy of Disruption of Cell Intrinsic Checkpoint Regulator CTLA-4 in Chimeric Antigen Receptor T Cells

Antonio Carusillo
Modulation of DNA Repair Pathways by HDR-CRISPR Promotes Seamless Genome Editing in Primary Human Hematopoietic Cells

Yue Du
Vectored Immunoprophylaxis for COVID-19 (COVIP)

Samuele Ferrari
Triggering P53 Activation and Trapping of Transcriptionally Active Recombinant AAV Sequences Are Inadvertent Consequences of HSC Genome Editing

Nayan Jain
CAR Design and Expression Determine Hyper-Proliferative States in TET2 Deficient T Cells

Francesco Manfredi
Exhausted, Tumor-Specific T Cells Can Be Exploited to Generate a Library of T-Cell Receptors for Adoptive T Cell Therapy in Blood Malignancies

Megan Manson
Optimization of Transcutaneous Ultrasound Mediated Gene Delivery into Large Animals

Katharina Meijboom
CRISPR/Cas9-Mediated Excision of ALS/FTD-Causing Hexanucleotide Repeat Expansion in C9ORF72 Rescues Major Disease Mechanisms

Helena Meyer-Berg
Generation of a Human 3D Lung Model for Therapeutic Gene Editing in Surfactant Protein B Deficiency

Alessio Nahmad
In-Vivo Engineered B Cells Retain Memory and Secrete High Titers of Anti-HIV Antibodies in Mice

Eleni Panagioti
Immunostimulatory Bacterial Antigen-Armed Oncolytic Measles Virotherapy Significantly Increases the Potency of Anti-PD1 Checkpoint Therapy

Sandhya Sharma
Memory Enriched Epstein-Barr Virus {EBV} Specific T-Cells with Broader Target Antigen Repertoire for the Treatment of EBV+ Malignancies

Toloo Taghian
Real-Time MR Tracking of AAV Gene Therapy with Enzyme-Activated MR Probes

Maria Vitale
An Oncolytic Adenoviral Vector Expressing an Anti-PD-L1 scFv Reduces Tumor Growth in a Melanoma Mouse Model

Jesse Weber
Capturing and Characterizing Single Cell Allelic Heterogeneity of CRISPR-Cas9 Gene Editing In Vivo

Cheyne Kurokawa, Mayo Clinic
The Innate Immune System as a Determinant for Response to Measles Virotherapy

Annicka Evans, Rice University
Characterization of a Protease-Activatable Adeno-Associated Virus Vector for Disease-Targeted Gene Delivery

Megan Keiser, PhD, Children’s Hospital of Philadelphia
Translating RNAi for Huntington’s Disease: Intra-Putaminal Delivery of AAV2/1.Mihds1 for Comprehensive Dosing, Biodistribution, Silencing and Safety in a Non-Human Primate

Bence Gyorgy, MD, PhD, Harvard Medical School
Allele-Specific Deafness Gene Disruption through Discrimination of a Single Base Change by S. aureus Cas9KKH Prevents Progressive Hearing Loss after AAV Mediated Gene Delivery

Mario Mietzsch, PhD, University of Florida
Structure-Function Characterization of Non-Primate AAV Capsids for Their Usage as Therapeutic Gene Delivery Vectors

Claire Domenger, PhD, University Hospital
Post-Transcriptional Fine-Tuning of AAV Vector Gene Expression for Hemophilia A Gene Therapy

Paul Maciocia, MBChB, BSc, University College London
A Simple Protein-Based Method for Generation of 'Off the Shelf' Allogeneic Chimeric Antigen Receptor T-Cells

Adele Ricciardi, Yale University
In Utero Gene Correction Mediated by PNA-Nanoparticles

Angelo Amabile, San Raffaele Telethon Institute for Gene Therapy
Inheritable Silencing of Endogenous Gene by Hit-and-Run Targeted Epigenetic Editing

Maria Carmina Castiello, San Raffaele Telethon Institute for Gene Therapy
Lentiviral-Mediated Gene Therapy Restores B Cell Homeostasis and Tolerance in Wiskott-Aldrich Syndrome Patients

Mei Dai, Cincinnati Children’s Hospital Medical Center
Platelets Transfusion New Role as Brain Therapeutics for Acute Neuronopathic Gaucher Disease

Adele Mucci, Hannover Medical School
Murine iPSC-Derived Macrophages Improve the In Vivo Disease Phenotype of Pulmonary Alveolar Proteinosis Due to Csf2rb Deficiency

Christopher Nelson, Duke University
Local and Systemic Gene Editing in a Mouse Model of Duchenne Muscular Dystrophy

Mohammadsharif Tabebordbar, Harvard University
In Vivo DMD Gene Editing in Muscles and Muscle Stem Cells of Dystrophic Mice

Garrett Berry, University of North Carolina-Chapel Hill
Pharmacological Regulation of Vesicular Trafficking as a Strategy to Enhance Recombinant AAV Transduction

Mari Hirvinen, University of Helsinki
Boosting the Immunogenicity of an Oncolytic Vaccinia Virus By Expression of DAI Can Enhance Anti-Tumor Immunity in Humanized Mice

Ulrike Jung, Beckman Research Institute of City of Hope
A Novel RNAi Trigger Design Retains Potent, Target Specific Activity Despite Emerging Mutations in the Target Site

Shruthi Naik, Mayo Clinic
Safety, Toxicity and Efficacy of Systemic Recombinant VSV Therapy in Pet Dogs With Spontaneous Cancer

Lauren Toth, Duke University
A Light-Inducible CRISPR/Cas9 System for Control of Endogenous Gene Activation

Erika Zonari, San Raffaele Telethon Institute for Gene Therapy (TIGET)
Hematopoietic Stem Cell Gene Therapy (2.0) Based on Purified CD34+CD38- Cells

Swaroopa Guda, Boston Childrens Hospital
Optimization of Lentivirus Vector RNA Polymerase II Driven microRNA Embedded shRNAs for Enhanced Processing and Efficient Knockdown of Bcl11a for Induction of Fetal Hemoglobin in Erythroid Cells

Juliette Hordeaux, University of Nantes
Long-term Neurologic Correction in the Pompe Disease Mice by Intrathecal Gene Therapy

Sharon Lam, Baylor College of Medicine
Ex Vivo Expanded Multi-Specific Cytotoxic T Lymphocytes Derived from HIV+ Patients and HIV Negative Donors Using GMP Compliant Methodologies Suppress HIV Replication

Michelle Marcus, Northwestern Univeristy
Engineering Exosomes as Therapeutic Delivery Vehicles

Nobuhiro Nishio, Nagoya University Hospital
Combined Armed Oncolytic Virus and CAR-T Cells Counter the Hostile Environment of Solid Tumors

Lukasz Swiech, Broad Institute
CRISPR-Mediated Genome Editing in the Mammalian Brain

Luca Biasco, San Raffaele Telethon Institute for Gene Therapy
Comprehensive Study of Lentiviral [quot]Integrome[quot] in WAS Gene Therapy Patients

Sourav Choudhury, University of Massachusetts Medical School
Identification of Novel Vectors Capable of CNS Transduction in Adult Mice after Single Round Selection Using DNA Shuffled AAV Capsid Library

Drew Deniger, University of Texas MD Anderson Cancer Center
Clinical Implications of ROR1-Specific T Cells That Target B-Cell Leukemia

Charles Lai, Massachusetts General Hospital
Non-Invasive In Vivo Imaging, Biodistribution and Clearance Analyses of Intravenously Administered Extracellular Vesicles

Rajiv Sharma, Children's Hospital of Philadelphia
In Vivo ZFN Mediated Targeting of Albumin as a Platform for Expression of Multiple Therapeutic Genes

Barbara Smith, University of Florida
Acid alpha-Glucosidase Gene Replacement Therapy to the Diaphragm in Ventilator-Dependent Pompe Disease: One-Year Respiratory Motor Outcomes

Anthony S. Fargnoli, University of Pennsylvania
Molecular Cardiac Surgery Mediated ssAAV1-SERCA2a Gene Therapy Restores Global and Regional Contractility in Ovine Ischemic Cardiomyopathy

Alice Giustacchini, San Raffaele Telethon Institute for Gene Therapy
microRNA 126 Is a New Regulator of Hematopoietic Stem Cells Homeostasis

Patrick J. Hanley, Baylor College of Medicine
Phase I Study To Improve Virus-Specific Immune Reconstitution after Cord Blood Transplantation Using Cord Blood-Derived Virus-Specific T Cells

Nicholas W. Keiser, University of Iowa
CFTR Gene Correction of Only the Surface Airway Epithelium Restores Antimicrobial Activity to CFTR Knockout Ferret Tracheas Ex Vivo

Rajiv Sharma, Children's Hospital of Philadelphia
Correction of Hemophilia B Phenotype Following ZFN Mediated Genome Editing in Adult Mice

Korashon L. Watts, Fred Hutchinson Cancer Research Center
Transfusion Independence and Rapid Engraftment of Nonhuman Primate Cord Blood Cells Expanded with the Combination of HOXB4 and Delta Ligand


26th Annual Meeting

May 16-20 | Los Angeles, CA

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