South Wales Echo

PRACTISE EASY GRATITUDE

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RATHER than rushing into the shower when your alarm goes off, pause and ease yourself into the day. Happiness consultant Samantha Clarke, speaking on behalf of Dorset Cereals (dorsetcere­als.com) suggests trying a 10 to 20 minute meditation.

Samantha recommends using an app like Calm (calm.com) or Headspace (headspace.com) to guide you through the process. If you’re really short of time, just listening to a soothing playlist on your commute can help relaxation.

HAVE A DIGITAL DETOX

MANAGING your use of technology can be a real time-maximiser in the morning. Life coach Danielle North recommends switching your phone for an alarm clock, so you can keep your devices turned off overnight.

She explains: “Once the phone is off, it’s easier to not grab it first thing.

“Perhaps you’ll even wait until you leave the house to switch it on? Either way, this habit can really help to ease stress and anxiety as well as lower your daily phone usage.”

FUEL THE BODY

NUTRITIONI­ST Rosie Mullen says: “The body is fasting as we sleep so when we wake we need to fuel it correctly to give you energy for the morning.”

Many studies have linked eating breakfast to good health, including better memory and concentrat­ion, energy levels and reduced risk of diabetes.

ENJOY THE LITTLE THINGS

HAVING a moment to yourself is important.

“Taking as little as 10 minutes to yourself will set your mindset for the rest of the day,” says anxiety expert Chloe Brotheridg­e.

Whether it’s taking a warm shower and appreciati­ng the water on your skin, lighting a candle or taking the scenic walk to work, having this time to create a sense of balance will have a positive effect on the hours that follow.

GENES are the building blocks of life – but like all things, they can sometimes go wrong, resulting in a range of conditions and diseases. Repairing or replacing these genes with ‘good’ ones, however, could solve or treat the problem, and this is what the emerging science of gene therapy is all about.

It was first suggested in the early-1970s that using ‘good’ DNA (genes are short sections of DNA) to replace defective DNA could treat inherited diseases, and since then scientists have been trying to work out how to do it, both for inherited conditions and many others.

The British Society for Gene and Cell Therapy (bsgct.org) says the first approved human gene therapy took place in 1990, on four-year-old Ashanti DeSilva who had ADA-SCID – an inherited disease that prevents normal developmen­t of the immune system. The therapy meant the little girl no longer needed to be kept in isolation and could go to school.

“When the human genome was mapped nearly 20 years ago, the notion that it could potentiall­y unlock therapies capable of fixing genes responsibl­e for some of the world’s most devastatin­g diseases was an idea of the future,” says gene therapy expert Professor Bobby Gaspar, speaking on behalf of Jeans for Genes Day, the annual campaign for Genetic Disorders UK (geneticdis­ordersuk.org).

“We are at the forefront of a new era of treatment for genetic diseases using gene and cell therapies. Some are one-time, potentiall­y curative investigat­ional therapies that could provide life-changing benefits to patients and their families.”

Prof Gaspar says there are currently more than 10 cell and gene therapy products approved in the EU, ranging from products that treat cancer to rare immune deficienci­es.

A number of these are approved in the UK and available on the NHS in specialise­d centres, and with nearly 3,000 clinical gene therapy trials underway worldwide, the number of treatments is expected to grow significan­tly over the next few years.

Here, Prof Gaspar – a professor of paediatric­s and immunology at the UCL Great Ormond Street Institute of Child Health and chief scientific officer at Orchard Therapeuti­cs, a gene therapy company that seeks to permanentl­y correct rare, often-fatal diseases – outlines five ways gene therapy can cure or slow a disease...

CANCER

“A VARIETY of efforts are underway to use gene therapy to treat cancer. Some types of gene therapy aim to boost the body’s immune cells to attack cancer cells, while others are designed to attack the cancer cells directly.

“One way the body protects itself from cancer is through T-cells, a main component of the immune system. But some cancers are good at avoiding these protection mechanisms,” says Prof Gaspar.

“Chimeric antigen receptor, or CAR T-cell therapy, is a new form of immunother­apy that uses specially altered T-cells to more specifical­ly target cancer cells. Some of the patient’s T-cells are collected from their blood, then geneticall­y modified to produce special CAR proteins on the surface.

“When these CAR T-cells are reinfused into the patient, the new receptors help the T-cells identify and attack cancer cells specifical­ly and kill them.”

VISION

“THERE are more than 250 genetic mutations that can lead to a type of blindness called inherited retinal diseases, or IRD.

“People with a defect in the RPE65 gene start losing their vision in childhood. As the disease progresses, patients experience gradual loss of peripheral and central vision, which can eventually lead to blindness.

“Gene therapy for some IRD patients became available in 2017, delivering a normal copy of the RPE65 gene directly to the retinal cells at the back of the eye using a naturally-occurring virus as a delivery vehicle.”

MOVEMENT

“FOR children with the genetic disorder spinal muscular atrophy, or SMA, a rare muscular dystrophy, motor nerve cells in the spinal cord are damaged, causing patients to lose muscle strength and the ability to walk, eat or even breathe,” says Prof Gaspar.

“SMA is caused by a mutation in a gene called SMN which is critical to the function of the nerves that control muscle movement. Without this gene, those nerve cells can’t properly function and eventually die, leading to debilitati­ng and often fatal muscle weakness.

“Researcher­s recently developed the first US-approved gene therapy to treat children less than two years of age with SMA. The therapy is designed to target the cause of SMA by replacing the missing or nonworking gene with a new, working copy of a human SMN gene, helping motor neuron cells work properly.”

INFECTIOUS DISEASES

“RESEARCHER­S believe targeted gene therapy and gene editing may have widespread applicatio­n for a range of infectious diseases that aren’t amenable to standard clinical management, including HIV.

“Although HIV isn’t a hereditary disease, the virus does live and replicate in DNA,” Prof Gaspar explains. “Another early but encouragin­g approach uses a gene editing technology combined with a new long-acting, antiretrov­iral treatment to suppress HIV replicatio­n and eliminate HIV from cells and organs of infected animals.

“Gene editing is an approach that precisely and efficientl­y modifies the DNA within a cell. In this approach, gene editing can ‘knock out’ a receptor called CCR5 on immune cells used by HIV to enter and invade cells. Without CCR5, HIV may no longer invade and cause disease.”

RARE DISEASES

“ONE approach being investigat­ed for a number of rare, often-fatal diseases uses gene-modified blood stem cells with a goal of permanentl­y correcting the underlying cause of disease.

“Blood stem cells are taken from the patient, corrected outside the body by introducin­g a working copy of the gene into the cells, then put back into the patient to potentiall­y cure the disease.

“Gene-modified blood stem cells have the capacity to self-renew and, once taken up in the bone marrow, can potentiall­y provide a lifelong supply of corrected cells. Because of their ability to become many different types of cells in the body, this approach has the potential to provide a lasting treatment for many different severe and often life-limiting inherited disorders, many of which have no approved treatment options available,” says Prof Gaspar.

“For instance, ADA-SCID, sometimes referred to as ‘bubble baby syndrome’, is a disease where babies lack almost all immune protection, leading to frequent and devastatin­g infections. Left untreated, babies rarely live past two years of age.

“Standard treatment options are not always effective or can carry significan­t risks. In 2016, the European Medicines Agency approved Strimvelis, a blood stem cell gene therapy for the treatment of ADA-SCID. Strimvelis was the first approved ex vivo gene therapy product in Europe.

“Jeans for Genes Day helped fund some of the earliest work using this type of gene therapy at Great Ormond Street Hospital in 2002, when Rhys Evans, a little boy with SCID, became one of the first children worldwide to be treated by gene therapy.”

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 ??  ?? Gene therapy expert Professor Bobby Gaspar
Gene therapy expert Professor Bobby Gaspar
 ??  ?? Don’t rush. Take time for breakfast
Don’t rush. Take time for breakfast

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