UNSW Sydney scientist Professor Merlin Crossley. Credit: UNSW In a landmark study that could lead to new therapies for sickle cell anaemia and other blood disorders, UNSW Sydney-led researchers have used CRISPR gene editing to introduce beneficial natural mutations into blood cells to boost their production of foetal haemoglobin. The research solves a 50-year-old mystery...
Category: <span>Genetics</span>
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CRISPR enhances gene therapy to fight inherited diseases
Shown is a transmission electron microscope image of adenovirus particles. A new study, in mice, combined molecular tools — adenovirus and CRISPR — to improve gene therapy. Credit: CDC/G. William Gary Jr Gene therapy has shown promise in treating inherited genetic diseases, but a major issue that has frustrated scientists remains: Replacing a “bad” gene...
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Complete genome sequence can be ID’d from amniotic fluid
(HealthDay)—The complete genome sequence of fetuses can be elucidated from amniotic fluid, according to a study published online March 15 in Clinical Chemistry. Qing Mao, from Complete Genomics Inc., in San Jose, Calif., and colleagues isolated cellular DNA and cell-free DNA (cfDNA) from the cell pellet of 31 amniocenteses and conducted sequencing to approximately 50× genome...
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Genome therapy could lead to new treatment for life-threatening blood disorders
Genome therapy with beneficial natural mutation could lead to new treatment for life-threatening blood disorders By introducing a beneficial natural mutation into blood cells using the gene-editing technique CRISPR, a UNSW Sydney-led team of scientists has been able to switch on production of foetal haemoglobin – an advance that could eventually lead to a cure for sickle...
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A new era in the interpretation of human genomic variation
In a commentary published today in Genetics in Medicine, Heidi Rehm, PhD, highlights the pressing need for standardized human genomic variant interpretation and calls on more stakeholders to join the data sharing movement. “It is time we considered the sharing of variant interpretations a fundamental right of patients to ensure that they receive accurate clinical care,”...
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Two new genes linked to Alzheimer’s risk
A team of researchers led by Cardiff University has identified two genes that influence a person’s risk of developing Alzheimer’s disease. The new finding, which builds on the team’s previous work of identifying 24 susceptibility genes, enables a better understanding of the mechanisms underlying the disease and offers further hope in developing new treatments. Dr...
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Large multi-ethnic study identifies many new genetic markers for lupus
Scientists from an international consortium have identified a large number of new genetic markers that predispose individuals to lupus The study is published in the July 17 issue of the journal Nature Communications and was led by researchers at Wake Forest Baptist Medical Center, Oklahoma Medical Research Foundation, King’s College of London and Genentech Inc. Autoimmune diseases...
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How Does the New ‘Gene-Altering’ Therapy Fight Cancer?
A new type of cancer treatment that involves altering a person’s genes — and could save children’s lives — passed a major hurdle this week, when a U.S. Food and Drug Administration (FDA) panel recommended that the agency approve the therapy, The New York Times reported. But how does the treatment work? The treatment is for...
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Key immunological mechanism for regulating intestinal flora discovered
Researchers at the Hospital del Mar Medical Research Institute (IMIM) have shown for the first time that immunoglobulin M, secreted by the human intestine, plays a key role in maintaining the diversity of intestinal flora by including and maintaining microorganisms that are beneficial to health. These results have been published in the journal Immunity. “We have...
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Breathing in a new gene therapy to treat pulmonary hypertension
Mount Sinai has partnered with Theragene Pharmaceuticals, Inc. to advance a novel airway-delivered gene therapy for treating pulmonary hypertension (PH), a form of high blood pressure in blood vessels in the lungs that is linked to heart failure. If the therapy succeeds in human clinical trials, it will provide patients for the first time with...