Treating sickle cell disease with gene therapy

After nearly two years of debate about its possible benefits and risks, the gene editing technique is now here to stay. An article in the December 27, 2015 edition of the Sunday Observer told of the first recorded use of the inexpensive CASPR-Cas9 gene editing technology to cut and ‘splice out’ bad genes and replace them with healthy genes.

What doctors say ‘cured’ sickle cell anemia patient

In a world first, a teenager with sickle cell disease achieved complete remission after an experimental gene therapy at Necker Children’s Hospital in Paris, researchers say. People with sickle-cell disease, a group of inherited blood disorders, have abnormal hemoglobin in their red blood cells, causing blood to clog in the tiny vessels and organs of the body.

Post doctoral fellow in Neuroscience

We aim to understand broad range of pathologies using molecular biology, behavioural and pharmacological methods. We are interested in candidates with strong background in at least one of the following fields; human genetics/epigenetics, human/clinical studies, drosophilia genetics, neuroanatomy or bioinformatics.

20 years after Dolly

Everything you always wanted to know about the Dolly the cloned sheep and what came next, from a biomedical researcher and cloning expert. It’s been 20 years since scientists in Scotland told the world about Dolly the sheep , the first mammal successfully cloned from an adult body cell .

Billions at stake in battle over gene-editing patents

Much like a surgical scalpel, the technique allows the genome to be edited by clipping out a specific area of DNA and in some cases replacing it with new instructions The first skirmish was fought last week in what could be a long war over a revolutionary patent on gene-editing technology, with colossal amounts of money at stake. Facing off are the top international experts in the fast-growing field of gene-editing – pitting American scientist Feng Zhang, against the French-American duo of Emmanuelle Charpentier and Jennifer Doudna.

Research institute retains gene editing patent rights

The Broad Institute, a biological and genomic research centre affiliated with MIT and Harvard, will keep valuable patents on a revolutionary gene-editing technology known as CRISPR, a U.S. patent agency ruled recently.a The U.S. Patent and Trademark Office’s Patent Trial and Appeal Board in Alexandria, Virginia, rejected a claim by a rival team, associated with the University of California at Berkeley and the University of Vienna in Austria, that it invented the technology first.a The patent rights could be worth billions of dollars because the technology could revolutionize treatment of genetic diseases and the genetic modification of crops.a Intellia Therapeutics Inc., which has a licensing deal with the University of California, said it would work on legal strategy with the University of California but that it was too early to comment on next steps.a The … (more)

Genome-editing patents belong to Broad Institute, board says

The US patent office ruled Wednesday that hotly disputed patents on a revolutionary genome-editing technology belong to the Broad Institute of Harvard and MIT. The US patent office ruled Wednesday that hotly disputed patents on a revolutionary genome-editing technology belong to the Broad Institute of Harvard and MIT, dealing a blow to the University of California’s efforts to overturn those patents.

Genome-editing patents belong to Broad Institute, board says

The US patent office ruled Wednesday that hotly disputed patents on a revolutionary genome-editing technology belong to the Broad Institute of Harvard and MIT. The US patent office ruled Wednesday that hotly disputed patents on a revolutionary genome-editing technology belong to the Broad Institute of Harvard and MIT, dealing a blow to the University of California’s efforts to overturn those patents.

Rare blood disease improves the defence against germs

Researchers of the HZI and of the University of Magdeburg find increased immune reaction associated with a rare bone marrow disease Patients afflicted by myeloproliferative neoplasia – a group of chronic malignant bone marrow diseases – bear a mutation in their haematopoietic stem cells. The mutation leads to the bone marrow producing too many blood cells, which thickens the blood.

Biotech leaders blast Trump’s immigration order

More than 160 leaders of biotechnology companies signed a strong letter of opposition to President Trump’s travel ban, arguing the ”poorly conceived and implemented” action strikes at the heart of an industry that originated in America, has been dominated by American companies, and is at the cutting edge of medicine. The letter, published Tuesday by the journal Nature Biotechnology, reveals that scientists abroad are afraid to come to the United States and are canceling trips.

Search goes on for bone marrow match for Alastair

ANDREW and Judy Kim are still searching the globe for a donor for their two-year-old son after he was diagnosed with a rare genetic condition. Mr and Mrs Kim launched an appeal for help in September but the search is still on for a matching donor and their son still needs hospital treatment.

Doctoral Students in Molecular Medicine

Aiming to combine complementary strengths in the Partnership, each institute brings a unique set of expertise, skills, and facilities encompassing the recognized research strengths of the is an international research institute focusing on human genomics and personalised medicine at the Helsinki Institute for Life Science of the University of Helsinki. integrates molecular medicine research and technology centre and biobanking infrastructure under one roof promoting translational research in grand challenge projects, specifically, the impact of genome information from the Finnish population in personalised health and medicine, individualised cancer medicine.

Doctoral Students in Molecular Medicine

Aiming to combine complementary strengths in the Partnership, each institute brings a unique set of expertise, skills, and facilities encompassing the recognized research strengths of the is an international research institute focusing on human genomics and personalised medicine at the Helsinki Institute for Life Science of the University of Helsinki. integrates molecular medicine research and technology centre and biobanking infrastructure under one roof promoting translational research in grand challenge projects, specifically, the impact of genome information from the Finnish population in personalised health and medicine, individualised cancer medicine.

Rat-grown mouse pancreases help reverse diabetes in mice

Mouse pancreases grown in rats generate functional, insulin-producing cells that can reverse diabetes when transplanted into mice with the disease, according to researchers at the Stanford University School of Medicine and the Institute of Medical Science at the University of Tokyo. The recipient animals required only days of immunosuppressive therapy to prevent rejection of the genetically matched rather than lifelong treatment.

Researchers reveal ‘alien’ life using synthetic DNA

Researchers at the Scripps Research Institute in La Jolla, California, introduced DNA molecules not found in nature to a common bacterium The E. coli bugs are able to grow and reproduce as normal despite containing two extra letters in their genetic code In the future the research could lead to creation of microbes capable of manufacturing entirely new proteins that could be used in medicine Some people are worried that the rapid advance of ‘synthetic biology’ could lead to the worrying prospect of new life-forms escaping from labs The modified E.coli bacteria was made by scientists who introduced DNA molecules not found in nature to a common bacterium.

Genome Engineering Staff Researcher – Hsu Laboratory

Research in the Hsu lab takes place at the intersection of bioengineering, genomics, and neuroscience to develop novel genome engineering technologies and uncover biological mechanisms behind neurodegenerative diseases and cancer. The Hsu Lab is recruiting to develop and apply novel technologies for precise and efficient manipulation of genetic and cell states.

Scientists reprogram embryonic stem cells to expand their potential

Researchers from UC Berkeley have found a way to reprogram mouse embryonic stem cells so that they exhibit developmental characteristics resembling those of fertilized eggs, or zygotes. While typical embryonic stem cells are unable to make tissues like the placenta, which allow the embryo to communicate with the mother, blocking a microRNA unleashes the potential to make these extra-embryonic tissues .

Johns Hopkins researchers helping in search for elusive cancer causing genes

A group of scientists at Johns Hopkins University is studying various bioinformatics methods to discover the ones that help identify the genetic mutations that encourage tumor cells to thrive in an effort to develop drugs to fight the cancer. A group of scientists at Johns Hopkins University is studying various bioinformatics methods to discover the ones that help identify the genetic mutations that encourage tumor cells to thrive in an effort to develop drugs to fight the cancer.

Teaching Computers to Recognize Sick Guts: Machine-Learning and the Microbiome

A new proof-of-concept study by researchers from the University of California San Diego succeeded in training computers to “learn” what a healthy versus an unhealthy gut microbiome looks like based on its genetic makeup. Since this can be done by genetically sequencing fecal samples, the research suggests there is great promise for new diagnostic tools that are, unlike blood draws, non-invasive.

Secretive Biotech Unicorn Moderna Unveils Pipeline, Financials

Moderna Therapeutics Inc., one of the best-funded private biotechnology companies in the U.S., unveiled its long-secret research and development pipeline on Monday, including experimental vaccines, cancer treatments and a cardiovascular therapy. The Cambridge, Massachusetts-based startup has about $1.3 billion in cash on hand from investors, pharmaceutical partners and government grants, according to a presentation Monday at the J.P.Morgan Chase & Co.

Amicus Therapeutics Provides Full-Year 2017 Strategic Outlook and Financial Guidance

Amicus Therapeutics , a global biotechnology company at the forefront of therapies for rare and orphan diseases, today provided its full-year 2017 strategic outlook and financial guidance. “In 2016 we made significant progress in our transformation to a global commercial-stage biotech company while we continued to advance and expand our tremendous pipeline of first- and/or best-in-class medicines for people living with devastating rare diseases,” stated John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc. “We begin 2017 in an excellent position to develop and deliver great medicines for patients and to create significant shareholder value.

Pluristem Therapeutics And New York Blood Center To Collaborate In…

Pluristem Therapeutics And New York Blood Center To Collaborate In Evaluating PLX-R18 As An Adjuvant Therapy To Umbilical Cord Blood Transplantation, Winning $900,000 Funding From BIRD Prior research has suggested that PLX-R18 helps cord blood cells to engraft more rapidly and effectively Grant of $900,000 from Israel-U.S. Binational Industrial Research and Development Foundation to fund the research Constitutes a 3rd potential hematologic indication for PLX-R18 HAIFA, Israel, Dec. 27, 2016 — Pluristem Therapeutics Inc. , a leading developer of placenta-based cell therapy products, today announced that it will collaborate with the New York Blood Center on preclinical studies of its Placental eXpanded -R18 cells to enhance the efficacy of umbilical cord blood transplantation.

Director of the Primate Genetics Research Section

The successful applicant will conduct both independent and collaborative research with a focus on genomic medicine or statistical genetics. In addition, this position will oversee the Primate Genetics Service Unit which provides services to support the genetic and genomic characterization of the nonhuman primate population and colony genetic management, as well as biostatics and bioinformatics services.