Friday, September 22, 2017

Manuela Corti, assistant professor in the department of pediatrics at the University of Florida in Gainesville, was awarded an MDA research grant totaling $298,954 over three years to study gene therapy in Friedreich’s ataxia (FA).

Source: MDA.org, 09/21/2017.

Our objective is to develop a treatment strategy for FA, one of the most common forms of ataxia. Specifically, our research plan focuses on the correction of both the cardiac and neurological degeneration found in the disease. These changes are due to harmful changes in the frataxin gene.
This work will specifically answer important mechanistic questions in a new FA mouse model, which has many of the symptoms of the human patients. First, we will identify the best route of delivery for the frataxin gene in the nervous system by comparing three different strategies for injecting the vector (delivery vehicle). Second, we will test the safety of repeated delivery of the frataxin gene vector in combination with medications that will prevent reactions against the frataxin protein and the vector components. Completion of this project will be an important milestone in the development of a treatment strategy that will dramatically improve quality of life for FA patients.

Bioavailability of resveratrol: Possibilities for enhancement

Konrad de Vries, Morné Strydom, Vanessa Steenkamp, Journal of Herbal Medicine, Available online 12 September 2017, ISSN 2210-8033, doi:10.1016/j.hermed.2017.09.002.

Resveratrol is a naturally occurring polyphenol that has been shown to elicit a variety of beneficial effects in vitro. Translating these gains to in vivo and clinical settings has proven to be a major challenge, because of its poor oral bioavailability. This caveat was confirmed after reviewing clinical trials conducted on this investigational product over the past two years. This review provides alternative methods of administration of resveratrol which may enhance its bioavailability. However, these methods: remain to be validated.
From current literature, it is clear that orally administered resveratrol has low bioavailability in vivo. A variety of methods that could overcome the inherent issues with resveratrol bioavailability have been proposed, however these need to be further validated in order to determine which are safe, effective and superior to traditional oral administration of resveratrol before clinical evaluation can take place.