This inventor holds 1 USPTO granted patent. Top assignee: Other. Active years: 2002.
Data source: USPTO (United States Patent and Trademark Office) public patent records. Weekly synchronization. How IDiyas builds this profile
Company Filing History:
1 out of 834,659 Years Active: 2002
Title: Paola Leone: Innovator in Gene Therapy Delivery Systems
Introduction
Paola Leone is a prominent inventor based in Philadelphia, PA (US). She has made significant contributions to the field of gene therapy, particularly in developing innovative delivery systems for therapeutic applications. Her work focuses on creating safe and effective methods for delivering genes to the brain.
Latest Patents
Paola Leone holds a patent for a "Delivery system for gene therapy to the brain." This gene delivery system is designed to ensure long-term expression throughout the brain. The invention utilizes a lipid-entrapped, polycation-condensed DNA (LPD) system for brain gene delivery, employing an adeno-associated virus (AAV) vector. The AAV plasmid developed by Leone is shown to be more effective than non-ITR containing plasmids in vivo. The results indicate that the LPD-AAV plasmid complexes can efficiently transduce neurons, with gene expression persisting for over 10 months in the brain. Additionally, the intraventricular delivery method combined with systemic hyperosmolality facilitates global gene delivery. This innovative approach has the potential to express the human aspartoacyclase (ASPA) gene in children with metabolic disorders over extended periods, demonstrating functional activity.
Career Highlights
Throughout her career, Paola Leone has focused on advancing gene therapy technologies. Her research has led to breakthroughs in the safe delivery of genetic material to the brain, which is crucial for treating various neurological conditions. Leone's dedication to her work has positioned her as a leading figure in the field of gene therapy.
Collaborations
Paola Leone has collaborated with notable colleagues, including Matthew J During and Frank L Sorgi. These partnerships have contributed to the success of her research and the development of her innovative gene delivery systems.
Conclusion
Paola Leone's contributions to gene therapy through her innovative delivery systems have the potential to transform treatment options for neurological disorders. Her work exemplifies the importance of research and innovation in advancing medical science.
Data source: USPTO (United States Patent and Trademark Office) public patent records. Weekly synchronization. How IDiyas builds this profile