NYSCF in the News
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.
 
 
 
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 
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. 
 
 
 
Wednesday, 07 December 2016 15:58

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.  
 
 
Wednesday, 07 December 2016 15:46

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 - Druckenmiller Fellow Alumnus Dr. Fabien Lafaille, The Rockefeller University, and NYSCF - Druckenmiller Fellow Alumnus and NYSCF - Robertson Stem Cell Investigator Alumnus Dr. Gabsang Lee, Johns Hopkins University School of Medicine, authored a paper discussing the use of induced pluripotent stem cells to explore why patients with familial dysautonomia exhibit differing disease severities despite identical genetic mutations. 
 
Published in Nature Medicine, the scientists created induced pluripotent stem cells from patient skin samples, and then turned these stem cells into the neurons affected by the disease. While the genetic mutations were identical amongst different patients, the derived neurons exhibited differences in survival and specification. 
 
This study demonstrates that induced pluripotent stem cell disease modeling accurately captures the differences in disease severity, presenting an important step towards personalized medicine. 
 
 
The Society for Neuroscience named NYSCF - Robertson Neuroscience Investigator Dr. Kay Tye as the recipient of the 2016 Young Investigator Award for her exceptional work studying the fundamentals of how the human brain interprets pain and pleasure. Dr. Tye is an Assistant Professor at the Massachusetts Institute of Technology Picower Institute for Learning and Memory.
 
Established in 1983, the $15,000 Young Investigator Award recognizes the outstanding achievements and contributions of a young neuroscientist who has recently received an advanced professional degree. The award was presented at the Society for Neuroscience annual meeting.
 
 
At some point in our lives, we or the people we love become patients. The results of the 2016 election will not change that fact. But they may well have a major impact on the funding of stem cell research, our nation’s commitment to advanced research, and our ability to pursue the highest and best science free of any political obstacles.  
 
The New York Stem Cell Foundation was created in 2005 at an exceptionally difficult time for stem cell research, when the federal government explicitly banned the use of government funds to support embryonic stem cell research. NYSCF, as a privately funded organization, played a key role in leading the way out of that difficult period for science. Our independence and private support since then have given us the freedom to pursue the most advanced research, irrespective of politics, and to build a broad constituency for the most advanced science. 
 
Over the past eleven years, NYSCF has made tremendous progress and helped to elevate the entire field of stem cell research. We are hopeful that the broad coalition of both public and private support for stem cell research that we have seen over the last eight years will not be lost. Today, NYSCF remains independent as an institution and regardless of the political climate, our only priority is to do everything we can to assure that the search for better treatments and cures continues to advance unencumbered by politics. 
 
Thank you for your ongoing support of NYSCF and stem cell research.
 

Susan L. Solomon
CEO and Co-Founder
The New York Stem Cell Foundation

 

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