In conjunction with the Alzheimer’s Disease Resource Center (ADRC), NYSCF presented their latest Alzheimer’s research at the Jewish Center of the Hamptons in East Hampton. Presentations by NYSCF CEO Susan L. Solomon, NYSCF Vice President for Stem Cell Research Dr. Scott Noggle, and President of ADRC Mary-Ann Ragona explored all aspects of Alzheimer’s disease, treatment, and research.
In addition, NYSCF Leadership Council member Carol Roaman graciously hosted NYSCF scientists and guests at a cocktail reception at her house in East Hampton. Guests mingled and spoke with NYSCF scientists, learning about the latest advances in stem cell research for many disease areas such as multiple sclerosis, bone engineering, and Parkinson’s disease among many others.
NSYCF – Druckenmiller Fellow Alumnus Dr. Marco Seandal of Weill Cornell Medical College published his latest research on how paternal age effects genetic mutations in Stem Cell Reports.
The scientists' experiments showed enhanced fitness of sperm stem cells with a specific age-related mutation. In addition, the scientists model will be useful for testing many different age-related genetic mutations carried by sperm to reveal mechanisms of disease.
Researchers at the Harvard Stem Cell Institute, led by NYSCF Chief Scientific Officer Dr. Kevin Eggan, discovered a new, promising treatment for ALS, or Lou Gehrig's disease. The research leading to this discovery began eight years ago and was initially and continuously supported by NYSCF.
Published in Science Translational Medicine, the scientists showed that genetically intervening in a biological pathway associated with ALS resulted in an increased survival rate of 5-10% in an animal model.
Critically, the scientists confirmed that this biological pathway is identical in animal models and in the human disease cells in a dish, forwshadowing the potential future elimination of animal model testing for ALS and other diseases.
Nature recently featured a profile on the education, background, and dynamic cross-discipline work of NYSCF Scientist Dr. Giuseppe Maria de Peppo. Dr. de Peppo focuses his research on engineering bone from stem cells using his background in technology and induced pluripotent stem cell research. His work developing the first ever vascularized bone tissue from stem cells was published last year in the Proceedings of the National Academy of Sciences of the USA and highlighted in the Wall Street Journal and other news outlets globally.
NYSCF - Robertson Stem Cell Investigator, Dr. Alex Meissner of Harvard University, mapped the methyl and demethylation patterns in human cells before and after fertilization. While this has previously been done extensively in mice, human DNA methylation patterns and changes are not fully understood.
The research, published in Nature, describes full genome DNA maps of methylation patterns in human cells during preimplantation development and embryonic stem cell derivation. The scientists confirmed that paternal genome demethylation is a general attribute of early mammalian development.
NYSCF - Robertson Stem Cell Investigator Dr. Paola Arlotta, of Harvard University, published her latest work on gene co-regulation in Nature Neuroscience.
The regulatory logic, or rules, that orchestrate the expression of the unique combinations of genes in each neuron class is unknown. In this study the scientists discovered that Fezf2 is the first selector gene able to regulate on its own the expression of large batteries of genes that collectively define corticospinal motor neurons (CSMN). CSMN are one specific class of cortical neurons responsible for the initiation and fine execution of motor function.These neurons are affected in diseases like ALS and are injured in spinal cord injury.
This is the first discovery of a selector gene for any class of neurons of the cerebral cortex and will aid efforts to program CSMN from stem cells in order to model disease affecting CSMN in vivo.
NSYCF - Robertson Neuroscience Investigator Dr. Ed Boyden, of MIT Media Lab, published his recent work in Nature Neuroscience. The paper describes the modification of a bacteria to respond to red wavelengths of light, named Jaws, and the use of Jaws to silence neural activity in the brains of awake mice when activated. This is the first light-sensitive molecule able to silence neurons noninvasively, potentially paving the way for use in humans to treat epilepsy and other neurological disorders.
The New York Times Magazine featured NYSCF’s cutting-edge research and our efforts to bring to patients our technique of preventing the transmission from mother to child of mitochondrial diseases as a cover story. The technique, pioneered and developed at the NYSCF Research Institute lead by NYSCF scientist Dr. Dieter Egli and his colleagues, involves transferring into a donor egg the nucleus of an egg cell from a woman affected by or with a family history of mitochondrial diseases. This would allow her to have a genetically related child that would be unaffected by this group of diseases. Dr. Egli presented this work to an FDA advisory committee earlier this spring in an important first step toward bringing this technique to patients.
There are currently no cures for this group of devastating diseases. Mitochondrial disorders affect approximately 1 in 10,000 people, while nearly 1 in 200 individuals carry mutant mitochondria. Symptoms, which most often appear in childhood, may include stunted growth, kidney disease, muscle weakness, neurological disorders, loss of vision and hearing, and respiratory problems, among others.
Our experiments have demonstrated the efficacy and safety of this technique, making NYSCF and our clinical collaborators at Columbia University the leading candidate to bring this technique to human clinical trials in the United States.