REGENERATIVE MEDICINE – Page 39 – REGENHEALTHSOLUTIONS (RHS)

PIER MARIA FORNASARI
2019-09-23

Optimized CRISPR guide RNA design for two high-fidelity Cas9 variants by deep learning

Source https://www.nature.com/articles/s41467-019-12281-8#Sec14 Highly specific Cas9 nucleases derived from SpCas9 are valuable tools for genome editing, but their wide applications are hampered by a lack of knowledge governing guide RNA (gRNA) activity. Here, we perform a genome-scale screen to measure gRNA activity for two highly specific SpCas9 variants (eSpCas9(1.1) and SpCas9-HF1) and wild-type SpCas9 (WT-SpCas9) in

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PIER MARIA FORNASARI
2019-09-23

Gene Editing Tool Gets Sharpened by WFIRM Team

Wake Forest Institute for Regenerative Medicine scientists have fine-tuned their delivery system to deliver a DNA editing tool to alter DNA sequences and modify gene function. The improved “hit and run” system works faster and is more efficient. “With this new method we are able to package together in one lentiviral capsid both essential components

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PIER MARIA FORNASARI
2019-09-22

Researchers from University of Regina develop a promising new ‘Trojan horse’ treatment for multiple sclerosis

We often take our immune and nervous systems for granted. We assume that our immune system will protect us from diseases and when pathogens invade our body. Likewise, we assume that our nervous system will take information from the environment, relay it to our brain and then allow our brain to move muscles. But what

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PIER MARIA FORNASARI
2019-09-22

Saving lives faster: Monash develops world-first laser incubator for blood

Monash University researchers, along with industry partner Haemokinesis, have developed the world’s first blood incubator using laser technology. This technology can slash blood incubation time to just 40 seconds, compared to the current industry gold standard of five minutes. The study shows that laser incubation can improve pre-transfusion testing for millions of patients undergoing blood

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PIER MARIA FORNASARI
2019-09-21

In Mice: Transplanted Brain Stem Cells Survive Without Anti-Rejection Drugs

By exploiting a feature of the immune system, researchers open the door for stem cell transplants to repair the brain. In experiments in mice, Johns Hopkins Medicine researchers say they have developed a way to successfully transplant certain protective brain cells without the need for lifelong anti-rejection drugs. A report on the research, published Sept. 16 in

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PIER MARIA FORNASARI
2019-09-21

World’s First Gene Therapy for Glycogen Storage Disease Produces Remarkable Results

At the Association for Glycogen Storage Disease’s 41st Annual Conference, Dr. David Weinstein of UConn School of Medicine and Connecticut Children’s presented his groundbreaking, one-year clinical trial results for the novel gene therapy treatment for glycogen storage disease (GSD). The rare and deadly genetic liver disorder, GSD type Ia, affects children from infancy through adulthood, causing dangerously

No Comments InGENE THERAPY RARE DISEASES

PIER MARIA FORNASARI
2019-09-20

New insight as to how cells maintain their identity

In the body’s cells, some proteins are of vital importance as to which genes are active or turned off. Now, researchers from the University of Copenhagen and the Memorial Sloan Kettering Cancer Center have discovered which proteins are necessary in order to maintain the proper genetic regulation. All of the more than 200 different cell

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PIER MARIA FORNASARI
2019-09-20

Bluebird bio Presents Updated Data from Phase 2/3 Clinical Study of Lenti-D™ Gene Therapy for Cerebral Adrenoleukodystrophy (CALD) at the 13th European Pediatric Neurology Society (EPNS) Congress

Long-Term Follow-up Data Show That the 88% of Patients Treated in the Starbeam Study (ALD-102) Were Free of Major Functional Disabilities (MFDs) at Two Years, and Continued to Remain MFD-Free at up to Five Years of Follow-up bluebird bio, Inc. (Nasdaq: BLUE) today announced updated results from the clinical development program for its investigational Lenti-D™ gene

No Comments InGENE THERAPY RARE DISEASES

PIER MARIA FORNASARI
2019-09-20

Medical Board of California meeting on stem cell clinics emphasized the rigorous stem cell-related clinic trials ongoing and in development and the risks coming from deviating from standard clinical trial and GMP practices.

Source The Niche Knoepfler Lab Stem Cell Blog This afternoon Dr. Knoepfler attended and gave public comment at the Medical Board of California stem cell task force meeting on unproven stem cell clinics. There were about 40 people attending. In part this meeting is a consequence of the national governing organization of state medical boards, the Federation of

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PIER MARIA FORNASARI
2019-09-20

Cancer-Related Mutations Identified in Primed and Naive Human Pluripotent Stem Cells

Source Cell Stem Cell Human pluripotent stem cells (hPSCs) are known to harbor chromosomal aberrations, affecting their tumorigenic potential. We established a strategy to identify cancer-related point mutations in hPSCs, detecting recurrent mutations in over 20 genes, alongside those previously detected in p53. Importantly, naive hPSCs harbor, on average, four times more mutations than their

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Optimized CRISPR guide RNA design for two high-fidelity Cas9 variants by deep learning

Gene Editing Tool Gets Sharpened by WFIRM Team

Researchers from University of Regina develop a promising new ‘Trojan horse’ treatment for multiple sclerosis

Saving lives faster: Monash develops world-first laser incubator for blood

In Mice: Transplanted Brain Stem Cells Survive Without Anti-Rejection Drugs

World’s First Gene Therapy for Glycogen Storage Disease Produces Remarkable Results

New insight as to how cells maintain their identity

Bluebird bio Presents Updated Data from Phase 2/3 Clinical Study of Lenti-D™ Gene Therapy for Cerebral Adrenoleukodystrophy (CALD) at the 13th European Pediatric Neurology Society (EPNS) Congress

Medical Board of California meeting on stem cell clinics emphasized the rigorous stem cell-related clinic trials ongoing and in development and the risks coming from deviating from standard clinical trial and GMP practices.

Cancer-Related Mutations Identified in Primed and Naive Human Pluripotent Stem Cells