NYSCF in the News & Research Spotlight

New Therapy Target Identified for Obesity Disorder
New Therapy Target Identified for Obesity Disorder
A team of researchers at Columbia University Medical Center including NYSCF - Robertson Investigator Dr. Dieter Egli have discovered that a specific enzyme deficiency in the brain is linked to most of the abnormalities in Prader-Willi syndrome, a rare genetic condition that causes extreme hunger and severe obesity beginning in childhood. The researchers used induced pluripotent stem cells, made from skin samples of patients with Prader-Willi, to create the neurons affected and identify the deficient enzyme. The enzyme, prohormone covertase, captures the link between the well known genetic mutation leading to Prader-Willi and the symptoms of the syndrome. 
The discovery, published in the Journal of Clinical Investigation, provides insight into the molecular mechanisms underlying the syndrome and highlights a novel target for drug therapy. This research has large implications not only on exciting new treatment targets and approaches for Prader-Willi patients, but also on the treatment and managment of obesity in general.
The Cell Therapy Glossary - An Essential Resource
The Cell Therapy Glossary - An Essential Resource
Susan L. Solomon, NYSCF CEO, and Dr. Michael Yaffe, NYSCF Vice President of Scientific Programs, contributed eleven new definitions and an editorial on the importance of including the patient voice in developing stem cell therapies to the third edition of the Cell Therapy Glossary. This latest edition includes over 35 new terms as selected by an expert panel on which Ms. Solomon and Mr. Yaffe sat.
Published in Regenerative Medicine, the Glossary serves a critical purpose for both expert scientists and the lay public by standardizing and explaining the cell therapy terminology used in the regenerative medicine industry. The Glossary allows scientists around the world to “speak the same language” when describing exciting updates and new research.
The terms contributed by NYSCF:
Haplotype – a group of genes or alleles usually inherited together from a single parent, reflecting the haploid genotype
HLA Haplobank – a panel or repository of iPSC lines that are homozygous for HLA types and could be used to derive immunologically-compatible tissues for therapeutic transplantation
Mitochondria – subcellular organelles that generate chemical energy to power cellular processes and also serve as sites for numerous metabolic processes and reactions
Mitochondrial replacement – a potential therapy to prevent certain mitochondrial diseases by replacing mutant maternal mitochondria in an oocyte (egg) with those from an unaffected individual prior to in vitro fertilization
Genomic modification – stable, intentional and directed change of a cell’s DNA sequence using biotechnology methods
CRISPR/Cas – a biotechnology tool, utilizing components of a prokaryotic immune system, for making highly specific genomic modifications
RNA-Seq – an analytical method that can reveal the identities and quantities of RNAs expressed from genes in a cellular sample
Disease-in-a-dish – a human disease model comprised of cultured cells displaying properties of diseased tissue
Note: many such models are generated using cells differentiated from iPSCs derived from individuals with a specific disease
Paracrine – influence or signaling by a cell on nearby cells or tissues through localized secretion and diffusion of small molecules or proteins
Investigational New Drug Application (IND) – a key step in development of a new drug or medical treatment in which the US Food and Drug Administration (FDA) is notified that a novel therapeutic will be used experimentally
Stem Cell Tourism – seeking or receiving stem-cell based treatments for disease or injury from clinics or practitioners that offer untested or unproven therapies 
Visual Stimulation Treats Alzheimer's Plaques in Mice
Visual Stimulation Treats Alzheimer's Plaques in Mice
NYSCF - Robertson Neuroscience Investigator Dr. Ed Boyden and his team at MIT Media Lab published their latest work on an exciting new, noninvasive approach to treating Alzheimer’s disease. Using LED lights flickering at a specific frequency, the researchers have shown that they can substantially reduce the beta amyloid plaques, hallmark neurological build-ups seen in Alzheimer’s disease, in the visual cortex of mice.
Described in Nature, this technique appears to work by inducing brain waves known as gamma oscillations, which the researchers discovered help the brain suppress beta amyloid production and invigorate cells responsible for destroying the plaques.
This research represents a potential breakthrough in the understanding and treatment of Alzheimer’s disease, a devastating disorder affecting millions around the world. 
New Tools for Simultaneous, Multi-Gene Editing
New Tools for Simultaneous, Multi-Gene Editing
NYSCF - Robertson Stem Cell Investigator and NYSCF - Robertson Stem Cell Prize Awardee Dr. Feng Zhang, the Broad Institute of Harvard and MIT, published his latest work expanding, modifying, and improving the CRISPR gene editing system.
The research, published in Nature Biotechnology, describes the ability to create CRISPR RNA that can be used to simplify multi-target genome editing. The scientists demonstrated this new tool by editing four genes in mammalian cells and three genes in the mouse brain, simultaneously. 
Gene editing technologies have opened up an entirely new arm of scientific research. The ability to modify multiple genes at one time is extremely useful for researching and ultimately, treating complex multi-gene conditions and diseases.  
Steps Towards Engineering Biological Cascades
Steps Towards Engineering Biological Cascades

NYSCF - Robertson Stem Cell Investigator Dr. Ed Boyden and his team at MIT Media Lab published their latest work on how to modify genetic circuits. The research, published in Nature Chemistry, describes the successful creation of synthetic biology cascades within liposomes, structures within cells, an important step towards engineering biological reactions for a variety of functions. 


Read the paper in Nature Chemistry >>

  • NYSCF Press Releases

    Partnership Results in the Availability of Fully-Sequenced Stem Cell Lines
    A collaboration beginning in 2013 between the NYSCF Research Institute and the Personal Genome Project (PGP) resulted in the availability of a unique new stem…

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    Feng Zhang Receives 2016 NYSCF – Robertson Stem Cell Prize
    NYSCF announced today that Feng Zhang, PhD, is the 2016 recipient of the NYSCF – Robertson Stem Cell Prize for his pioneering advances to edit…

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    NYSCF Announces the Six Members of the 2016 Class of NYSCF - Robertson Investigators
    NYSCF announced the 2016 class of NYSCF – Robertson Investigators, welcoming six of the most talented stem cell researchers and neuroscientists from around the world…

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    NYSCF Calls For Faster FDA Review to Curb Stem Cell Tourism Citing Baby Born Using Mitochondrial Replacement Therapy
    A report in The New Scientist presents claims that the first baby was born in Mexico by a New York based doctor using mitochondrial replacement…

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    NYSCF Research Institute Teams Up With National Stem Cell Foundation to Advance Neurodegeneration Research
    The National Stem Cell Foundation (NSCF) and the NYSCF Research Institute announced that a grant from NSCF will be used to fund NYSCF research on…

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