Stem cell derived neurons for research relevant to Alzheimer's and Niemann-Pick type C diseases

December 9, 2009

Stem cell derived neurons may allow scientists to determine whether breakdowns in the transport of proteins, lipids and other materials within cells trigger the neuronal death and neurodegeneration that characterize Alzheimer's disease (AD) and the rarer but always fatal neurological disorder, Niemann-Pick Type C (NPC), according to a presentation that Lawrence B. Goldstein, Ph.D., of the University of California, San Diego, School of Medicine and Howard Hughes Medical Institute (HHMI) will give at the American Society for Cell Biology (ASCB) 49th Annual Meeting, Dec. 5-9, 2009 in San Diego.

In research using fruit flies, mice and human cell cultures as lab models, Goldstein pioneered the study of how early defects in the intracellular physical transport system may be the driving force behind severe neuronal dysfunction.

Using human (hESCs), Goldstein and his team have produced human neurons in which the NPC gene is switched off, providing the first close look at cellular transport in a human neuron lacking normal function of the gene.

With induced (IPS), Goldstein has derived human neurons representing the genetic "familial" form of AD as well as the far more common "sporadic" AD.

By comparing the biochemical and cellular makeup of these two types of stem cell derived neurons, Goldstein hopes to reveal how their known genetic differences affect their transport of vital cellular cargoes and other cellular behaviors.

Such research "may yield an understanding of what components of sporadic disease are defined by ," said Goldstein, professor in the Department of Cellular & Molecular Medicine, an HHMI investigator and director of UC San Diego's Stem Cell Program.

AD is now the seventh leading cause of death in the U.S., according to the National Centers for Health Statistics. The National Niemann-Pick Disease Foundation reports that children born with NPC rarely survive beyond the age of 20.

Source: American Society for Cell Biology

Explore further: Stem-cell therapies for brain more complicated than thought

Related Stories

Recommended for you

Genomes uncover life's early history

August 24, 2015

A University of Manchester scientist is part of a team which has carried out one of the biggest ever analyses of genomes on life of all forms.

Rare nautilus sighted for the first time in three decades

August 25, 2015

In early August, biologist Peter Ward returned from the South Pacific with news that he encountered an old friend, one he hadn't seen in over three decades. The University of Washington professor had seen what he considers ...

Study shows female frogs susceptible to 'decoy effect'

August 28, 2015

(Phys.org)—A pair of researchers has found that female túngaras, frogs that live in parts of Mexico and Central and South America, appear to be susceptible to the "decoy effect." In their paper published in the journal ...

Why a mutant rice called Big Grain1 yields such big grains

August 24, 2015

(Phys.org)—Rice is one of the most important staple crops grown by humans—very possibly the most important in history. With 4.3 billion inhabitants, Asia is home to 60 percent of the world's population, so it's unsurprising ...

0 comments

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.