A5011111049- Lysosomal Storage Disorders Research
- Spinal Dysraphism and Malformations
- Prenatal Screening and Diagnostics
- RNA Interference and Gene Delivery
- CRISPR and Genetic Engineering
- Neurogenetic and Muscular Disorders Research
- RNA Research and Splicing
- Virus-based gene therapy research
- Cellular transport and secretion
- interferon and immune responses
- Cardiovascular Disease and Adiposity
- Genetics and Neurodevelopmental Disorders
- Adipose Tissue and Metabolism
- Healthcare and Venom Research
- Climate Change and Health Impacts
- Plant-based Medicinal Research
- Intraocular Surgery and Lenses
- Gut microbiota and health
- Pharmacological Effects and Toxicity Studies
- Autoimmune and Inflammatory Disorders Research
- Epilepsy research and treatment
- MicroRNA in disease regulation
- Retinal and Optic Conditions
- Toxin Mechanisms and Immunotoxins
- Healthcare professionals’ stress and burnout
University of Florida
2014-2024
Florida College
2014-2024
University of Florida Health Science Center
2023
University of Florida Health
2023
Metal elements are essential components of approximately half all cellular proteins, and one-third known enzymes thus far metalloenzymes. Several proteins undoubtedly impact the transduction efficiency recombinant adeno-associated virus (AAV) vectors, but precise role metal ions in this process has not been studied detail. In present studies, we systematically evaluated effects 10 (calcium, cobalt, copper, iron, magnesium, manganese, molybdenum, potassium, sodium, zinc) on AAV vectors. We...
Abstract Mucopolysaccharidosis type IIIB (MPS IIIB) is an autosomal recessive lysosomal storage disease caused by mutations in the gene that encodes protein N-acetyl-glucosaminidase (NAGLU). Defective NAGLU activity results aberrant retention of heparan sulfate within lysosomes leading to progressive central nervous system (CNS) degeneration. Intravenous treatment options are limited need overcome blood–brain barrier and gain successful entry into CNS. Additionally, we have demonstrated AAV8...
Sanfilippo Syndrome Type-B remains an untreatable childhood neurodegenerative disease with great burden for both patient and caregiver. Very few clinical trials have been undertaken to treat the disease, none of these yet yielded clinically obtainable products patients. Caused by a simple enzyme function deficiency, has considered prospect gene-therapy interventions. Adeno-associated virus (AAV) major choice therapeutic gene delivery due its relatively low-immunogenicity, versatility tissue...
Background: Physician burnout is increasingly recognized as a problem in physician well-being and may negatively affect patient care outcomes. Burnout can begin at any point of training or practice, potentially early the first year medical school. Thus, there need to characterize possible students step optimizing strategies for mitigation. Traditionally, has been studied using survey-based variables; however, identifying novel physiological molecular biomarkers could allow expansion...
Abstract Gut microbiota are a community of organisms that colonize the intestinal tract with essential roles in immune function, gut epithelial integrity, and metabolite production. Dysbiosis occurs when environment is negatively altered causing detrimental changes to these functions, which may predispose an individual breast cancer development. To understand relationship between dysbiosis development, fecal samples were collected from 27 post-menopausal patients varying hormone receptor...
Abstract Introduction: Sanfilippo syndrome is a rare disease and fatal genetic disorder in the United States with no FDA-approved treatment, comprehensive assessment of economic burden available. Objective To develop model to estimate associated (US) using valued intangibles (disability-adjusted life years lost) indirect (lost caregiver productivity) from 2023 onward. Design Setting: A multistage comorbidity was generated publicly available literature on disability, 14 disability weights...
Abstract Introduction: Sanfilippo syndrome is a rare disease and fatal genetic disorder in the United States with no FDA-approved treatment, comprehensive assessment of economic burden available. Objectives: To develop model to estimate associated (US) using valued intangibles (disability-adjusted life years lost) indirect (lost caregiver productivity) from 2023 onward. Design Setting: A multistage comorbidity was generated publicly available literature on disability, 14 disability weights...
Abstract Introduction: Sanfilippo syndrome is a rare disease and fatal genetic disorder in the United States with no FDA-approved treatment, comprehensive assessment of economic burden available. Objectives: To develop model to estimate associated (US) using valued intangibles (disability-adjusted life years lost) indirect (lost caregiver productivity) from 2023 onward. Design Setting: A multistage comorbidity was generated publicly available literature on disability, 14 disability weights...
Abstract Background: Behavioral, social, and physical characteristics are posited to distinguish the sexes, yet research on transcription-level sexual differences in brain is limited. Here, we investigated sexually divergent transcriptomics prepubertal cynomolgus macaques, a commonly used surrogate species humans. Methods: A transcriptomic profile using RNA sequencing was generated for temporal lobe, ventral midbrain, cerebellum of 3 female male macaques previously treated with an...
Sanfilippo syndrome Type-B, also known as mucopolysaccharidosis IIIB (MPS IIIB), accounts for approximately one-third of all patients and is characterized by a similar natural history Type-A. Patients suffer from developmental regression, bone malformation, organomegaly, GI distress, profound neurological deficits. Despite human trials enzyme replacement therapy (ERT) (SBC-103, AX250) in MPS IIIB, there currently no FDA approved treatment few palliative options. The major concerns ERT gene...
Abstract Introduction: Sanfilippo syndrome is a rare disease and fatal genetic disorder with no FDA-approved treatment in the United States (US), comprehensive assessment of economic burden available. Objectives: To develop model to estimate associated US using direct costs, indirect costs valued intangibles (disability-adjusted life years, or DALYs) from 2023 onward. Design Setting: A multistage comorbidity was generated based on symptoms, disability weights 2010 Global Burden Disease...
Behavioral, social, and physical characteristics are posited to distinguish the sexes, yet research on transcription-level sexual differences in brain is limited. Here, we investigated sexually divergent transcriptomics prepubertal cynomolgus macaques, a commonly used surrogate species humans.A transcriptomic profile using RNA sequencing was generated for temporal lobe, ventral midbrain, cerebellum of 3 female male macaques previously treated with an Adeno-associated virus vector mix....