Publications by authors named "T R Flotte"
Background/objectives: α-1 antitrypsin (AAT) deficiency is an inherited, genetic condition characterized by reduced serum levels of AAT and increased risk of developing emphysema and liver disease. AAT is normally synthesized primarily in the liver, but muscle-targeting with a recombinant adeno-associated virus (rAAV) vector for α-1 antitrypsin (AAT) gene therapy has been used to minimize liver exposure to the virus and hepatotoxicity. Clinical trials of direct intramuscular (IM) administration of rAAV1-hAAT have demonstrated its overall safety and transgene expression for 5 years.
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- - Neurological diseases caused by single gene defects can be treated through AAV-mediated gene therapy, but delivering this therapy to the brain is difficult because of the blood-brain barrier.
- - Advanced techniques, like convection-enhanced delivery and image-guided methods to cerebrospinal fluid spaces, allow for precise gene therapy delivery to target specific brain areas.
- - Neuroimaging methods, including MRI and fMRI, are crucial for both delivering AAV vectors and monitoring the effectiveness of the gene therapy over time.
Background And Aims: Effective approaches for prevention of hepatocellular carcinoma (HCC) will have a significant impact on HCC-related mortality. There are strong preclinical data and rationale to support targeting epidermal growth factor receptor (EGFR) for HCC chemoprevention. Small molecule inhibitors of EGFR have been Food and Drug Administration-approved for cancer therapy, which provides an opportunity to repurpose one of these drugs for chemoprevention of HCC.
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