Press Releases, News and Articles 

NICE approves gene therapy for rare Bubble Baby Syndrome

Children with ADA-SCID, severe combined immunodeficiency due to adenosine deaminase deficiency, are extremely vulnerable to infection and usually live in isolation to minimise the risk, hence the nickname ‘baby in a bubble’. Up until now the only treatment has been a stem cell transplant but these are risky and it is not always possible to find a good match. Now NICE (National Institute for Health and Care Excellence) has approved a new treatment for those children who cannot find a good match. It could mean children with ADA-SCID have the chance of going to school and socialising without the constant fear of catching a simple infection that could prove life threatening. The full article is

Lack of support increases pregnant woman's biological age, shortening telomeres

New York: Low socioeconomic status during childhood and poor family social support appear to prematurely age pregnant women on a cellular level, potentially raising the risk for complications, a new study has found. For the study, the researchers examined blood from pregnant women to evaluate the length of telomeres -- structures at the end of chromosomes that are used by scientists as a measure of biological (as opposed to chronological) age. Read more at: http://zeenews.india.com/health/lack-of-support-may-increase-pregnant-womans-biological-age-2050720

Yescarta is the Second gene therapy approved by the FDA, targeting blood cancer

U.S. regulators on Wednesday approved a second gene therapy for a blood cancer, a one-time, custom-made treatment for aggressive lymphoma in adults. The Food and Drug Administration allowed sales of the treatment from Kite Pharma. It uses the same technology, called CAR-T, as the first gene therapy approved in the U.S. in August, a treatment for childhood leukemia from Novartis Pharmaceuticals. "In just several decades, gene therapy has gone from being a promising concept to a practical solution to deadly and largely untreatable forms of cancer," FDA Commissioner Dr. Scott Gottlieb said in a statement. The full article is available at the following link: http://abcnews.go.com/Technology/wire

Air Pollution Makes Babies' Cells Age Faster due to accelerated Telomere Shortening

There’s growing evidence that exposure to air pollution can have a number of unhealthy consequences, from cancer to heart disease and respiratory illnesses. In recent years, researchers have also linked air pollution exposure to faster aging in adult cells. In a new study published in JAMA Pediatrics, an international group of researchers conducted the first detailed look at pollution’s effect on developing babies in utero. They found that the more pollution expectant moms were exposed to while they were pregnant, the shorter their babies’ telomeres: parts of the DNA in every cell that act as a molecular clock keeping track of the cell’s age, and the body’s. The full article is available on

Kymriah™ is the First Ever Gene Therapy Approved by the FDA

On the 30th August 2017, the U.S. Food and Drug Administration issued a historic action making the first gene therapy available in the United States, ushering in a new approach to the treatment of cancer and other serious and life-threatening diseases. The FDA approved Kymriah (tisagenlecleucel) for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL).“We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer,” said FDA Commissioner Scott Gottlieb, M.D. “New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in o

The importance of good neighbours [to protect Telomeres]: Birds who live next to family or friends a

The lyrics to the much-loved soap, Neighbours, claim that 'everybody needs good neighbours' – and it seems that this may even apply to birds. Scientists have found that birds who live next to family or friends are physically healthier and age more slowly.The researchers hope their findings will provide exciting new information about how conflict over space and resources can be resolved in the animal kingdom. The full article is available at the following link: http://www.dailymail.co.uk/sciencetech/article-4956242/Birs-live-family-friends-healthier.html

FDA panel backs gene therapy for rare form of blindness

Spark Therapeutics Inc's experimental gene therapy for a rare form of blindness improves vision and should be approved, advisers to the Food and Drug Administration concluded on Thursday, paving the way for the first U.S. gene therapy for an inherited disease. The panel voted unanimously in favor of the treatment, Luxturna, which is designed to treat inherited retinal diseases caused by defects in a gene known as RPE65, which tells cells to produce an enzyme critical to normal vision. Read the full article at: http://wixx.com/news/articles/2017/oct/12/fda-panel-votes-in-favor-of-sparks-blindness-gene-therapy/

Gene therapy halts progression of cerebral adrenoleukodystrophy in clinical trial

Adrenoleukodystrophy — depicted in the 1992 movie “Lorenzo’s Oil” — is a genetic disease that most severely affects boys. Caused by a defective gene on the X chromosome, it triggers a build-up of fatty acids that damage the protective myelin sheaths of the brain’s neurons, leading to cognitive and motor impairment. The most devastating form of the disease is cerebral adrenoleukodystrophy (CALD), marked by loss of myelin and brain inflammation. Without treatment, CALD ultimately leads to a vegetative state, typically claiming boys’ lives within 10 years of diagnosis. But now, a breakthrough treatment is offering hope to families affected by adrenoleukodystrophy. A gene therapy treatment effec

Titia de Lange to receive 2017 Rosenstiel Award

Titia de Lange, a biochemist who studies the protective ends of chromosomes known as telomeres, has been named the 2017 recipient of the Lewis S. Rosenstiel Award for Distinguished Work in Basic Medical Research, a highly prestigious honor presented annually by Brandeis University. She is recognized for her elucidation of the mechanism of telomere protection and the maintenance of genome stability. de Lange, who is Leon Hess Professor and head of the Laboratory of Cell Biology and Genetics, is known for her discovery of the mechanisms by which telomeres repress DNA repair and damage responses that mistake chromosome ends for DNA breaks that need to be fixed. Her work has revealed how telomer

Gene therapy shows promise for reversing blindness

Most causes of untreatable blindness occur due to loss of the millions of light sensitive photoreceptor cells that line the retina, similar to the pixels in a digital camera. In a laboratory study in Oxford, researchers have shown how it might be possible to reverse blindness using gene therapy to reprogram cells at the back of the eye to become light sensitive. The full article is available at the following link: https://www.sciencedaily.com/releases/2017/10/171004084948.htm

Dr. Bill Andrews, Ph.D, explains the role of Telomeres and Telomerase in Aging on Korean National Te

Inside the nucleus of a cell, our genes are arranged along twisted, double-stranded molecules of DNA called chromosomes. At the ends of the chromosomes are stretches of DNA called telomeres, which protect our genetic data, make it possible for cells to divide, and hold secrets to how we age. Telomeres can be compared to the plastic tips of shoelaces, because they keep chromosome ends from fraying and sticking to each other, which would destroy or scramble an organism's genetic information. Yet, each time a cell divides, the telomeres get shorter. When they get too short, the cell can no longer divide; it becomes inactive or "senescent" or it dies. Dr. Bill Andrews,Ph.D, in this recent docume

World Heart Day

Heart disease is the leading cause of death and disability in several countries such as Malaysia, where heart disease remains as the number one cause of death for almost a decade (2005 – 2014). Similarly, in Singapore, heart disease is accountable for 29.5% of deaths in the nation in 2016 – translating to one out of three deaths related to heart failure. In conjunction with World Heart Day, we have compiled three new studies that aid in curbing the killer disease, one of them related to Telomere's lenght. The full article is available at the following link: https://today.mims.com/world-heart-day-treating-heart-failure-one-research-at-a-time

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