A5020499831- CRISPR and Genetic Engineering
- Prenatal Screening and Diagnostics
- RNA Interference and Gene Delivery
- Extracellular vesicles in disease
- Hemoglobinopathies and Related Disorders
- Viral Infectious Diseases and Gene Expression in Insects
- Biological Activity of Diterpenoids and Biflavonoids
- Retinal Development and Disorders
- CAR-T cell therapy research
- Retinoids in leukemia and cellular processes
- Cellular Mechanics and Interactions
- Cancer Treatment and Pharmacology
- Cellular transport and secretion
- Genetics, Aging, and Longevity in Model Organisms
St. Jude Children's Research Hospital
2022-2025
University of Tennessee Health Science Center
2015-2022
Genome editing enzymes can introduce targeted changes to the DNA in living cells 1-4 , transforming biological research and enabling first approved gene therapy for sickle cell disease 5 . However, their genome-wide activity be altered by genetic variation at on- or off-target sites 6-8 potentially impacting both precision therapeutic safety. Due a lack of scalable methods measure from large populations diverse target libraries, frequency extent these variant effects on remains unknown....
Sickle cell disease (SCD) is a common, severe genetic blood disorder. Current pharmacotherapies are partially effective and allogeneic hematopoietic stem transplantation associated with immune toxicities. Genome editing of patient cells (HSCs) to reactivate fetal hemoglobin (HbF) in erythroid progeny offers an alternative potentially curative approach treat SCD. Although the FDA released guidelines for evaluating genome risks, it remains unclear how best pre-clinical assessment genome-edited...
Pediatric osteosarcomas (OS) exhibit extensive genomic instability that has complicated the identification of new targeted therapies. We found vast majority 108 patient tumor samples and patient-derived xenografts (PDXs), which display an unusually dilated endoplasmic reticulum (ER), have reduced expression four COPII vesicle components trigger aberrant accumulation procollagen-I protein within ER. CRISPR activation technology was used to increase two these, SAR1A SEC24D, physiological...
Abstract Sickle cell disease (SCD) is a common severe blood disorder, caused by one major point mutation in the HBB gene. Current pharmacotherapies are only partially effective and potentially curative allogeneic hematopoietic stem transplantation (HSCT) associated with immune toxicities. Genome editing of autologous patient cells (HSCs) to reactivate fetal hemoglobin (HbF) erythroid progeny offers approach treat SCD circumvents some problems HSCT. Although FDA has released guidelines for...
Genetic vascular disorders are prevalent diseases that have diverse etiologies and few treatment options. Pathogenic missense mutations in the alpha actin isotype 2 gene (