Speeding up drug discovery for brain diseases
July 31, 2019
Whitehead Institute team finds drugs that activate a key brain gene; initial tests in cells and mice show promise for rare, untreatable neurodevelopmental disorder
Tag: Neurobiology
How to be a good host (for Zika virus)
April 22, 2019
Using a genome-wide CRISPR screen in neural progenitor cells, Whitehead Institute scientists uncover what the Zika virus needs to infect human cells
Only in your head
October 9, 2018
Researchers uncover how a ubiquitously expressed mutation causes a brain-specific disorder.
Stem cell-derived Zika model suggests mechanisms underlying microcephaly
June 19, 2018
Infected microglia cells can ferry Zika virus to developing brain
Network of diverse noncoding RNAs acts in the brain
June 7, 2018
First known network consisting of three types of regulatory RNAs is identified
Genetic body/brain connection identified in genomic region linked to autism
October 6, 2017
For the first time, Whitehead Institute scientists have documented a direct link between deletions in two genes—fam57ba and doc2a—in zebrafish and certain brain and body traits, such as seizures, hyperactivity, large head size, and increased fat content. Both genes reside in the 16p11.2 region of the genome, which has been linked to multiple brain and body disorders in humans, including autism spectrum disorder, developmental delays, seizures, and obesity.
Derived neural immune cells enable new facet of neurodegeneration research
September 26, 2016
Whitehead Institute scientists have devised a protocol for pushing human pluripotent stem cells to become microglia—the specialized immune cells that maintain the brain and care for it after injury. Microglia play an important role in neurodegenerative diseases, including Parkinson’s and Alzheimer’s, and studying these cells has been very difficult until now.
Identifying a genetic mutation behind sporadic Parkinson’s disease
April 20, 2016
Using a novel method, Whitehead Institute researchers have determined how mutations that are not located within genes are identified through genome-wide association studies (GWAS) and can contribute to sporadic Parkinson’s disease, the most common form of the condition. The approach could be used to analyze GWAS results for other sporadic diseases with genetic causes, such as multiple sclerosis, diabetes, and cancer.
New mouse-human modeling system enables study of disease development in vivo
January 25, 2016
Whitehead Institute researchers have created a new mouse-human modeling system that could be used to study neural crest development as well as the modeling of a variety of neural crest related diseases, including such cancers as melanoma and neurofibromatosis.
Enhanced-sensitivity NMR could reveal clues on how proteins fold
October 8, 2015
Until now, it has been difficult to fully characterize the different structures that proteins can take on in their natural environments. However, using a new technique known as sensitivity-enhanced nuclear magnetic resonance (NMR), Whitehead Institute and MIT researchers have shown that they can analyze the structure that a yeast protein forms as it interacts with other proteins in a cell.
Direct generation of neural stem cells could enable transplantation therapy
November 6, 2014
Induced neural stem cells (iNSCs) hold promise for therapeutic transplantation, but their potential in this capacity has been limited by failed efforts to maintain such cells in their multi-potent NSC state. Now, Whitehead Institute scientists have created iNSCs that remain in the multi-potent state—without ongoing expression of reprogramming factors. This allows the iNSCs to self-renew repeatedly to generate cells in quantities sufficient for therapy.
Combination therapy a potential strategy for treating Niemann-Pick disease
May 15, 2014
Whitehead Institute researchers have identified a potential dual-pronged approach to treating Niemann-Pick type C (NPC) disease, a rare but devastating genetic disorder. By studying nerve and liver cells grown from NPC patient-derived induced pluripotent stem cells (iPSCs), the scientists determined that although cholesterol does accumulate abnormally in the cells of NPC patients, a more significant problem may be defective autophagy—a basic cellular function that degrades and recycles unneeded or faulty molecules, components, or organelles in a cell. Here, the scientists propose two drugs, one to reduce cholesterol buildup and the other to induce autophagy, as a strategy for treating NPC.