NYSCF and the Beyond Batten Disease Foundation (BBDF) were selected as a national innovator by the Milken Institute and presented their unique multistakeholder collaboration to find cures and treatments for juvenile Batten disease at the 6th annual FasterCures Partnering for Cures conference in New York City.
BBDF and NYSCF will develop stem cell resources to investigate and explore new treatments and, ultimately, cures for this fatal childhood illness.
NYSCF - Robertson Stem Cell Investigator Dr. Derrick Rossi, of Harvard University, published the latest from his lab on using a gene editing technique to block HIV from invading and destroying the immune system. The researhc, published in Cell Stem Cell, describes how the scientists used CRISPR-Cas gene editing technology to edit genes out of human blood-forming stem cells, making them impervious to HIV.
While this research is, at minimum, years away from reaching the clinic, it proves a promising and exciting new direction for HIV treatments and cures.
NYSCF - Robertson Stem Cell Investigator Dr. Alex Meissner, of Harvard University, published his recent analysis of whole-genome buslfate sequencing (WGBS). This sequencing method allows scientists to analyze an entire genome for DNA methylation patterns, but it remains underutilized due to high costs.
Dr. Meissner and his team used reference data to analyze miminum sequencing requirements, as compared to whole genome sequencing. The scientists identified trade-offs with increasing or decreasing sequencing coverage in their report, published in Nature Methods.
NYSCF - Druckenmiller Fellow Dr. Sandra Pinho, of Albert Einstein College of Medicine, was part of a team of scientists who recently discovered new insights into blood-forming stem cell regulation. The scientists discovered that a non-blood forming type of cell, called a megakaryocyte, can directly regulate the amount of active blood-forming stem cells in mice through the release of a specific protein.
This research helps lend insight into the regulation, maintenance, and proliferation of blood-forming stem cells, a process that is not yet fully understood. Nature Medicine published the results of the study.
NYSCF CEO Susan L. Solomon responded to "The Trials of Stem Cell Therapy," published in The New York Times on September 15, 2014 with a Letter to the Editor, refuting the article's claim that stem cell research has not delivered on its early promises of creating personalized medicine. In her letter, Ms. Solomon cited just a few of the many medical breakthroughs discovered using stem cell research in the past few years, including NYSCF's development of a cure for mitochondrial diseases, which The New York Times Magazine featured in its cover story on June 29, 2014.
NYSCF – Druckenmiller Postdoctoral Fellow Dr. Raffaella Di Micco of New York University School of Medicine was first author for a paper published in Cell Reports identifying a key protein, implicated in many different cancers, that appears to play a pivotal role in keeping stem cells in an immature or pluripotent state, meaning the cells can become any type of cell in the body. This finding has key implications on cancer research and treatments and there are ongoing clinical trials targeting this protein.
NYSCF – Robertson Stem Cell Investigator Dr. Kristen Brennand of the Icahn School of Medicine at Mount Sinai led a team of scientists that discovered that neural cell models of schizophrenia, made using induced pluripotent cells derived from patients with schizophrenia, secrete increased amounts of three types of neurotransmitters commonly associated with many psychiatric disorders.
This research, published in Stem Cell Reports, could shed light into the chemical basis for this chronic and debilitating brain disorder.
The NYSCF Conference is unique: it focuses on translational stem cell research, demonstrating the potential to advance cures for the major diseases of our time.
It is designed for those with an interest in translational medicine, including researchers, clinicians, patient advocates, government and health officials, graduate students, and industry. The panels on Wednesday are designed to reach a broader audience, including the lay public.