Liposome Based Gene Therapy for Canavan Disease

The subject of neurodegenerative disease in children would seem, at first glance, to be a rather unlikely theme for a photographic exhibit. However, as a researcher in the field of neuroscience and gene therapy, nothing seems more natural to me than sharing the inspirational and moving views of humanity and medicine that I have had the privilege to see through my scientific work. Few things can compare to seeing our work put to use in helping sick children who are programmed to have a short life expectancy along with a poor quality of life. As both an amateur photographer and a medical scientist, I have a unique vantage point to share on the artistic and humanistic elements entwined in my work. Over the years in which I have been acting as one of the lead investigators for a project involving gene replacement for Canavan's Disease, I have personally witnessed the unique human drama in which this revolutionary medical advance has developed.

Although it is rare, Canavan Disease deserves greater public awareness, especially in the Jewish community where "carriers" (those with gene mutations that are not overtly manifest) can be identified. Canavan disease has a powerful human dimension that extends far beyond the people who are themselves affected, and is also of great scientific import in developing therapies that will eventually be directed toward other brain diseases.

...Pictures can say more than words alone.

Paola Leone 1999

Some of these photographs were displayed at the American Museum of Natural History, New York, NY. Genomic Revolution Exhibit, May 26th2001 -January 2nd, 2002 and Museum of Natural History, North Carolina. Genomic Revolution Exhibit, April 26th, January 2nd, 2003

All photos by Paola Leone

Slide 1	Slide 2
Slide 3	Slide 5
Slide 6	Slide 7
Slide 12	Slide 13
Slide 14	Slide 15
Slide 16	Slide17
Slide 18	Slide19
Slide 20

Images of Current AAV Mediated Gene Therapy Trial

Photos by Paola Leone

First Canavan patient that underwent brain gene therapy using adeno-associated viral vectors - June 2001. Helene Karlin holds Lindsay on her hospital discharge day.	Third Canavan patient that underwent brain gene therapy using adeno-associated viral vectors - July 2001. Richard Sontag kisses Jacob Sontag on his hospital discharge day.
Second Canavan Patient underwent brain gene therapy in July 2001. Max Randell smiles at his mother Ilyce on his discharge day.	The first surgery using adeno-associated viral vectors for a neurogenetic disorder: June 5th, 2001, Thomas Jefferson Hospital (Philadelphia) : Dr. Erol Veznedarouglu, Dr. Andrew Freese, Mr. McPhee, Dr. Christopher Janson, Dr. Meryl Latsko.