A5055278243- Virus-based gene therapy research
- Hemophilia Treatment and Research
- CAR-T cell therapy research
- Blood Coagulation and Thrombosis Mechanisms
- CRISPR and Genetic Engineering
- Viral Infectious Diseases and Gene Expression in Insects
- Coagulation, Bradykinin, Polyphosphates, and Angioedema
- Immune Cell Function and Interaction
- Viral gastroenteritis research and epidemiology
- Monoclonal and Polyclonal Antibodies Research
- Animal Virus Infections Studies
- Influenza Virus Research Studies
- Cytomegalovirus and herpesvirus research
- Bacteriophages and microbial interactions
- Respiratory viral infections research
- T-cell and B-cell Immunology
- Cancer-related gene regulation
- Platelet Disorders and Treatments
- Immunotherapy and Immune Responses
- Herpesvirus Infections and Treatments
- Chronic Myeloid Leukemia Treatments
- HIV Research and Treatment
- Transgenic Plants and Applications
Children's Hospital of Philadelphia
2016-2025
University of Pennsylvania
2011-2020
University of Arkansas for Medical Sciences
2020
The Wistar Institute
2010-2014
Base editors can correct disease-causing genetic variants. After a neonate had received diagnosis of severe carbamoyl-phosphate synthetase 1 deficiency, disease with an estimated 50% mortality in early infancy, we immediately began to develop customized lipid nanoparticle-delivered base-editing therapy. regulatory approval been obtained for the therapy, patient two infusions at approximately 7 and 8 months age. In weeks after initial infusion, was able receive increased amount dietary...
Influenza viruses cause severe illnesses and death, mainly in the aged population. Protection afforded by licensed vaccines through subtype-specific neutralizing antibodies is incomplete, especially when vaccine antigens fail to closely match those of circulating viral strains. Efforts are underway generate a so-called universal influenza expressing conserved sequences that induce broad protection multiple strains virus induction CD8⁺ T cells. Here we assess effect potent antiviral cell...
ABSTRACT In this study, we tested the effect of neutralizing Abs to different serotypes E1-deleted Ad vectors on immunogenicity homologous vector or a derived from heterologous serotype. Our results showed that, as expected, even low titers passively transferred significantly reduced vectors' ability elicit transgene-specific CD8+ T cell responses. addition, changed fate transgene product–specific cells by promoting their transition into central memory pool, which resulted in markedly...
Here we describe a series of replication-defective adenovirus vectors designed to express transgene products from two expression cassettes placed into the deleted E1 and E3 domains. Vectors that contained an cassette with cytomegalovirus promoter in forward orientation chicken β-actin reverse grew acceptable yields expressed both transgenes. Additionally, they elicited immune responses products. Levels vectors' immunogenicity were influenced by presence regulatory elements shared between...
Methamphetamine (METH) continues to be among the most addictive and abused drugs in United States. Unfortunately, there are currently no Food Drug Administration–approved pharmacological treatments for METH-use disorder. We have previously explored use of adeno-associated viral (AAV)-mediated gene transfer an anti-METH monoclonal antibody. Here, we advance our approach by generating a novel single-chain variable fragment (scFv)-Fc fusion construct (termed 7F9-Fc) packaged into AAV serotype 8...
Abstract A sizable proportion of hemophilia inhibitor patients fails immune tolerance induction and requires bypass agents for long-term bleed management. Recombinant human-activated coagulation Factor VII (rhFVIIa) is an on-demand hemostatic agent bleeds in patients. Prophylactic use rhFVIIa may enable sustained management patients, but the critical relationship circulating levels clinical outcome that setting remains unclear. To address this vivo, we used rat (HA) model exhibits...
Abstract Methamphetamine (METH) continues to be amongst the most addictive and abused drugs in US. Unfortunately, there are currently no FDA approved pharmacological treatments for METH substance abuse disorder. As an alternative approach, we have previously explored use of Adeno-associated viral (AAV) mediated gene transfer anti-METH monoclonal antibody. Here, advance our approach by generating a novel scFv-Fc fusion construct (7F9-Fc), packaged into AAV serotype 8 vector (called...