Giant Axonal Neuropathy News and Research

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Investigational gene therapy shows promise for rare childhood neurodegenerative disease

An investigational gene therapy for a rare neurodegenerative disease that begins in early childhood, known as giant axonal neuropathy (GAN), was well tolerated and showed signs of therapeutic benefit in a clinical trial led by the National Institutes of Health.

20 Mar 2024

UCLA researchers uncover dual role of GAN gene in neurological disorder, head and neck cancer

Researchers from the UCLA Health Jonsson Comprehensive Cancer Center show for the first time that a gene usually linked to giant axonal neuropathy, a rare and severe neurological condition, also plays a role in inhibiting aggressive tumor cell growth in head and neck cancers.

29 Feb 2024

Researchers discover molecular underpinnings of Alexander disease

Scientists have known that genetic mutations leading to the production of a defective protein called GFAP cause Alexander disease (AxD), a debilitating neurodegenerative condition that can present during infancy, adolescence, or adulthood.

21 Nov 2019

UC Davis researchers reveal how four proteins come together to help assemble tubulin

When they think about how cells put together the molecules that make life work, biologists have tended to think of assembly lines: Add A to B, tack on C, and so on. But the reality might be more like a molecular version of a 3-D printer, where a single mechanism assembles the molecule in one go.

21 Aug 2015

Innovative gene transfer-based treatment for kids with giant axonal neuropathy

University of North Carolina School of Medicine researchers have developed an innovative, experimental gene transfer-based treatment for children with giant axonal neuropathy (GAN).

26 Jun 2015

Researchers identify how a defective protein plays central role in Giant Axonal Neuropathy

A team of international researchers led by Northwestern Medicine scientists has identified how a defective protein plays a central role in a rare, lethal childhood disease known as Giant Axonal Neuropathy, or GAN.

29 Apr 2013