Tel Aviv University’s Professor Illana Gozes receives top RARE gene award

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Credit: American Friends of Tel Aviv University (AFTAU)

Tel Aviv University's Prof. Illana Gozes was awarded the 2016 RARE Champion of Hope — Science International Prize by Global Genes, a leading global advocacy non-profit organization for patients and families fighting rare and genetic diseases.

The ceremony took place at the 5th Annual Tribute to Champions of Hope presented by PRA Health Sciences on September 23 and 24 at the Huntington Beach Hyatt Resort and Spa in Aliso Viejo, CA. The annual blue carpet event honors and celebrates rare disease advocates, scientists, patients, and supporters.

Prof. Gozes is the incumbent of the Lily and Avraham Gildor Chair for the Investigation of Growth Factors; Head of the Elton Laboratory for Molecular Neuroendocrinology at TAU's Sackler Faculty of Medicine; and a member of TAU's Adams Super Center for Brain Studies and Sagol School of Neuroscience.

More than 350 individuals and organizations worldwide were nominated by members of the Global Genes Board of Directors and Medical and Science Advisory Board for their notable efforts in rare disease advocacy, science, collaborative sciences, and medical care and treatment. Only a few of the nominees were selected for the top honor.

A leader in genetic research

Prof. Gozes is a vanguard on the front of genetic research. She discovered the ADNP gene that causes the ADNP syndrome (also known as the Helsmoortal-Van Der AA syndrome), deregulates in Alzheimer's disease (AD) and in schizophrenia, and mutates in autism. Prof. Gozes has also been instrumental in drawing attention to the ways that ADNP binds to microtubules — tubes within nerve cells that maintain cell shape and serve as "train tracks" for movement of biological material through the brain.

"With her commitment to research and new therapies, Prof. Gozes continues to help many undiagnosed patients receive the therapies and tests needed to be happy, healthy, and diagnosed," according to Global Genes.

"There is no one else more worthy of receiving this award than Prof. Gozes," said Angela Downing, Chair of the ADNP Kids Research Foundation, who presented the award to Prof. Gozes. "She is a respected neuroscientist, a beloved professor and a mentor to many. She has worked tirelessly in her career not only for science but for humanity. Prof. Gozes delivers nothing but excellence in terms of research. Her commitment to our children, who suffer from ADNP disorders, is strong and we stand confident knowing she stands with us."

Sandra Sermone, Founder and President of the ADNP Kids Research Foundation, added: "I have known Prof. Gozes for the last two years, after my son was diagnosed with an ADNP mutation. She is a leader in ADNP research and drug development and works tirelessly to advance research and potential treatments for our children with this heart-breaking disease. She is committed, caring and compassionate. There is no one more deserving of an award associated with hope."

Praising a community

Global Genes is a leading global advocacy non-profit organization for patients and families fighting rare and genetic diseases. The organization hopes to find treatments and cures for the 7,000+ rare and genetic diseases that affect an estimated 30 million Americans and over 350 million people worldwide through building awareness, developing patient-focused educational tools, and funding patient care programs and early investigative research.

"I feel privileged to have helped this community," said Prof. Gozes. "I may have been able to help only a few families, but then they discussed the experience and passed on vital information to others in similar situations. Together, as a community, they have a voice which calls for more funding to support further critical research."

Together with Coronis Neurosciences (on agreement with [email protected] Aviv University), Prof. Gozes is currently developing an ADNP-based drug for cognitive impairment in schizophrenia. A small fragment of ADNP called davunetide has shown promise in Phase IIa clinical trials, and Prof. Gozes is harnessing it for further advanced trials on large cohorts of patients. The exposure to the knowledgeable and compassionate community of Global Genes and the honor of receiving the RARE Champion of Hope — Science International Prize will no doubt accelerate drug development.

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Tel Aviv University (TAU) is inherently linked to the cultural, scientific and entrepreneurial mecca it represents. It is one of the world's most dynamic research centers and Israel's most distinguished learning environment. Its unique-in-Israel multidisciplinary environment is highly coveted by young researchers and scholars returning to Israel from post-docs and junior faculty positions in the US.

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