NYSCF in the News
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


Leaders in stem cell biology and researchers at the forefront of translational medicine convened at the 2016 NYSCF Conference to discuss the latest advances in using stem cells to understand and treat human disease. Through presentations, panels, posters and numerous informal discussions, attendees gained exciting new insights into disease mechanisms and heard about novel approaches to treat a wide range of debilitating conditions.  
Key meeting highlights included updates on stem cell research on:
Macular Degeneration - Presentations by Drs. Masayo Takahashi, RIKEN-CDB, Japan, and Sally Temple, Neural Stem Cell Institute, on the initiation of clinical trials involving stem cell-based treatments for macular degeneration. 
Cancer - Drs. Isabelle Riviere, Memorial Sloan Kettering Cancer Center, Saar Gill, University of Pennsylvania, and Khalid Shah, Harvard University and Massachusetts General Hospital, shared the latest CAR-T and stem cell-based cancer therapies, an exciting and promising approach for patients around the world.
In addition, a keynote address featured Greg Simon, Executive Director of the Cancer Moonshot Taskforce, who discussed the President's initiative to use novel collaboration structures and a boost of funding to make giant research leaps against many different types of the disease.
Tissue Engineering - Novel approachs to treating complicated disease included a presentation by Dr. Tracy Grikscheit, Children’s Hospital Los Angeles and the University of Southern California, who discussed how her lab uses new approaches for intestine and colon repair via stem cell-based tissue engineering. 
Diabetes - Drs. Gerald Napom, Benaroya Research Institute, and Qizhi Tang, University of California San Francisco, debated strategies for overcoming immunological barriers in cell therapy for diabetes. 
Other presentations addressed heart disease, neurodegenerative disorders, bone repair and novel drug discovery platforms.
The NYSCF conference is unique, focusing on translational stem cell research demonstrating the potential to advance cures for the major diseases of our time. It is designed for all professionals with an interest in stem cell research, including physicians, researchers, clinical investigators, professors, government and health officials, postdoctoral fellows and graduate students. Stay tuned for information on The NYSCF Conference 2017! 
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