Abstract
Nanopharmaceutics Innovations in Gene Therapy: Moving Towards Non-Viral and Non-Invasive Delivery Methods
Highlights
The targeted insertion of DNA coding for a therapeutic gene into the nuclei of diseased cells or tissues followed by its expression, is one of the most promising new therapies for a host of diseases and conditions [1]
UniQure is developing the GDNF gene in their associated viral (AAV)-2 delivery vector (NCT01621581) and Oxford Biomedica reported Phase 1/2 clinical trial results with ProSavin, a tricistronic lentivirus-based vector encoding tyrosine hydroxylase, L-amino acid decarboxylase (AADC), and cyclohydrolase 1, both aimed at restoring dopamine production in patients with advanced Parkinson’s disease [3]
In spite of the tremendous progress, future challenges in gene therapy treatments include the development of gene delivery vectors with targeting ability, controllable, high and prolonged transfection efficiency, improved safety and less complicated or non-invasive administration methods
Summary
The targeted insertion of DNA coding for a therapeutic gene into the nuclei of diseased cells or tissues followed by its expression, is one of the most promising new therapies for a host of diseases and conditions [1]. More than 1,800 gene therapy clinical trials have been carried out in the past 25 years, including the currently in-progress of about 400 studies [2]. As recent reports of encouraging progress are emerging with viral vector-based therapies, the field is developing more confidence in gene therapy applications.
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