Category Archives: Gene editing

US regulators approve two gene therapies for sickle cell disease

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Blood disorder that can lead to premature death affects estimated 100,000 people in the United States, most of whom are Black

The US Food and Drug Administration has approved a pair of gene therapies for sickle cell disease, including the first treatment based on the breakthrough Crispr gene-editing technology, opening up two “transformative therapy” avenues for some patients.

The FDA approved Lyfgenia from Bluebird Bio, and a separate treatment called Casgevy by partners Vertex Pharmaceuticals and Crispr Therapeutics. Both therapies are made from the patients’ own blood stem cells and were approved for people aged 12 and older.

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Pets could be gene-edited under new English law, says RSPCA

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Act opens door to technology being used to create cats and dogs with extreme features, says charity

Pets could be subjected to gene editing under a new government act, the RSPCA has warned.

The animal charity has said that the Genetic Technology (Precision Breeding) Act applies to all vertebrate animals, not only farmed animals, and that it could lead to cats and dogs being gene-edited to include extreme features.

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Scientist convicted of editing babies’ genes has Hong Kong visa revoked over ‘false statement’

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Local media report inquiry launched over application by Chinese scientist He Jiankui, whose work sparked ethics storm

Hong Kong has reportedly revoked a visa for the controversial gene therapy scientist He Jiankui less than a day after it was revealed he’d been granted one, despite having a criminal record in China for illegal medical practices.

Hong Kong immigration officials said his visa was rescinded and a criminal investigation launched into allegations He had lied on his application form, the South China Morning Post reported.

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Scientist who edited babies’ genes says he acted ‘too quickly’

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Exclusive: He Jiankui stops short of apologising for procedure that shocked the world of science in 2018

The scientist at the heart of the scandal involving the world’s first gene-edited babies has said he moved “too quickly” by pressing ahead with the procedure.

He Jiankui sent shock waves across the world of science when he announced in 2018 that he had edited the genes of twin girls, Lulu and Nana, before birth. He was subsequently sacked by his university in Shenzhen, received a three-year prison sentence, and was broadly condemned for having gone ahead with the risky, ethically contentious and medically unjustified procedure with inadequate consent from the families involved.

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Gene-edited sheep offer hope for treatment of lethal childhood disease

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Roslin Institute engineered a flock to help research into the genetics of Batten disease

A flock of gene-edited sheep has been used by scientists to pinpoint a promising treatment for a lethal inherited brain disease that afflicts young children. The researchers, based in the UK and US, say their work could lead to the development of drugs to alleviate infantile Batten disease.

In the UK, Batten disease affects between 100 and 150 children and young adults and is inherited from two symptomless parents who each carry a rare recessive gene mutation.

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Fears environment bills could be sidelined amid Tory leadership race

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Campaigners warn crucial legislation must not be abandoned while the UK government is distracted

Crucial environment legislation must not be allowed to be sidelined or abandoned amid the distraction of a Tory leadership race, campaigners have warned.

Ministers openly admit they do not know what is going on with much of the legislation, but those who remain in government are working with skeleton teams to get bills in shape to be passed.

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Ageing reversal: scientists rejuvenate tissues in middle-aged mice

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Prospect of medical therapies that rewind clock for humans edges a little closer

The prospect of medical therapies that rewind the clock on the ageing process has edged a little closer after scientists safely rejuvenated tissues in middle-aged mice.

Researchers in the US treated healthy animals with a form of gene therapy that refreshed older cells, making the animals more youthful according to biological markers that are used to measure the effects of ageing.

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Why pig-to-human heart transplant is for now only a last resort

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Analysis: As doctors monitor world’s first human recipient of pig heart, safety and ethical concerns remain

The world’s first transplant of a genetically altered pig heart into an ailing human is a landmark for medical science, but the operation, and the approach more broadly, raise substantial safety and ethical concerns.

Surgeons at the University of Maryland Medical Center spent eight hours on Friday evening transplanting the heart from the pig into 57-year-old David Bennett, who had been in hospital for more than a month with terminal heart failure.

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Genetic engineering test with mosquitoes ‘may be game changer’ in eliminating malaria

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UK scientist says gene-drive study rendering female insects infertile may lead to ‘self destruct mosquito’ field tests within 10 years

Scientists have successfully wiped out a population of malaria-transmitting mosquitoes by using a radical form of genetic engineering to render the females infertile – in the most advanced and largest ever test of use of the technology to fight the disease.

As well as bringing fresh hope in the fight against one of the world’s biggest killers, the study lays the foundations for further trials of gene-drive technology, which could mean self-destroying mosquitoes being released into the wild within 10 years.

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He Jiankui, Chinese scientist who edited babies’ genes, jailed for three years

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The biophysicist was guilty of illegal practice in trying to alter the genetic makeup of twins Lula and Nana

The scientist who created the world’s first “gene-edited” babies has been sentenced to to three years in prison by a court in China.

He Jiankui said in November 2018 that he had used gene-editing technology known as Crispr-Cas9 to change the genes of twin girls, causing a backlash in China and globally about the ethics of his research and work.

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China gene-edited baby experiment ‘may have created unintended mutations’

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He Jiankui’s original research, published for the first time, could have failed, scientists say

The gene editing performed on Chinese twins to immunise them against HIV may have failed and created unintended mutations, scientists have said after the original research was made public for the first time.

Excerpts from the manuscript were released by the MIT Technology Review to show how Chinese biophysicist He Jiankui ignored ethical and scientific norms in creating the twins Lula and Nana, whose birth in late 2018 sent shockwaves through the scientific world.

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New gene editing tool could fix most harmful DNA mutations

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‘Prime editing’ more precise than Crispr-Cas9, but still needs time before use on humans

Scientists have raised fresh hopes for treating people with genetic disorders by inventing a powerful new molecular tool that, in principle, can correct the vast majority of mutations that cause human genetic diseases.

The procedure, named “prime editing”, can mend about 89% of the 75,000 or so harmful mutations known to mangle the human genome and lead to conditions such as cystic fibrosis, sickle cell anaemia, and a nerve-destroying illness called Tay-Sachs disease.

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First human-monkey chimera raises concern among scientists

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Researchers reprogrammed human cells before injecting them in the monkey embryo

Efforts to create human-animal chimeras have rebooted an ethical debate after reports emerged that scientists have produced monkey embryos containing human cells.

A chimera is an organism whose cells come from two or more “individuals”, with recent work looking at combinations from different species. The word comes from a beast from Greek mythology which was said to be part lion, part goat and part snake.

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One-off injection may drastically reduce heart attack risk

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Doctors hope to trial gene therapy on people with rare disorder in next three years

Doctors in the US have announced plans for a radical gene therapy that aims to drastically reduce the risk of heart attack, the world’s leading cause of death, with a one-off injection.

The researchers hope to trial the therapy within the next three years in people with a rare genetic disorder that makes them prone to heart attacks in their 30s and 40s. If the treatment proves safe and effective in the patients, doctors will seek approval to offer the jab to a wider population.

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Gene therapy could treat rare brain disorder in unborn babies

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Doctors could use Crispr tool to inject benign virus into foetus’s brain to ‘switch on’ key genes

Scientists are developing a radical form of gene therapy that could cure a devastating medical disorder by mending mutations in the brains of foetuses in the womb.

The treatment, which has never been attempted before, would involve doctors injecting the feotus’s brain with a harmless virus that infects the neurons and delivers a suite of molecules that correct the genetic faults.

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