Scientists create reprogrammable mouse in stem cell breakthrough, opening a new way to regenerate failing tissues in patients with diseases ranging from heart failure to diabetes

September 11, 2013 6:04 pm

Scientists create reprogrammable mouse in stem cell breakthrough

By Clive Cookson, Science Editor

Scientists in Spain have produced the world’s first embryonic stem cells within a live animal rather than in a laboratory dish. The experiment with mice at the National Cancer Research Centre in Madrid could open a new way to regenerate failing tissues in patients with diseases ranging from heart failure to diabetes. Therapeutic applications – for example to repair damaged spinal cord or make new insulin-producing cells – are distant. Even on the most optimistic assumptions clinical trials in people are unlikely to start in less in than five years, according to Dr Serrano.Therapy might involve inserting the reprogramming genes into a harmless virus that would carry them into diseased or damaged human tissues, where they would produce first stem cells and then rejuvenating specialist cells.

The researchers in Madrid adapted the technique for which Shinya Yamanaka of Japan won a Nobel Prizelast year. The Japanese team created embryonic stem cells of a new type – induced pluripotent stem cells or iPSCs – by reprogramming adult cells in a lab dish with a combination of four genes, which turned their clock back to an immature stage where they can become any sort of specialist cell.

The Spanish group bred genetically engineered mice with the same cocktail of four reprogramming genes – and switched them on by adding a particular drug to the animals’ drinking water.

After a few weeks, embryonic stem cells had appeared in multiple tissues and organs. The researchers extracted these cells and demonstrated through various tests that they were like those in a new embryo containing just 16 cells. This is an even earlier state of development than iPSCs made in vitro.

Embryonic cells made in “reprogrammable mice” also survive outside the animals in culture dishes. “So we can manipulate them in a laboratory,” said Maria Abad, lead author of a paper describing the discovery in the journal Nature. “The next step is studying if these new stem cells are capable of efficiently generating different tissues such as that of the pancreas, liver or kidney.”

“We can now start to think about methods for inducing regeneration locally and in a transitory manner for a particular damaged tissue,” added Manuel Serrano, head of the Madrid laboratory.

Writing a commentary in Nature, stem cell researchers George Daley and Alejandro De Los Angeles of Harvard University hailed the paper as “a landmark for what could become a powerful strategy in regenerative medicine: tissue reprogramming in situ”.

“The major challenge will be tightly controlling every step in this potential approach to treating patients in order to deliver clinical benefits, while avoiding potential complications,” said Chris Mason, professor of regenerative medicine at University College London. Ensuring safety will be a priority for clinical research based on the discovery, agreed Dr Serrano.

About bambooinnovator
Kee Koon Boon (“KB”) is the co-founder and director of HERO Investment Management which provides specialized fund management and investment advisory services to the ARCHEA Asia HERO Innovators Fund (www.heroinnovator.com), the only Asian SMID-cap tech-focused fund in the industry. KB is an internationally featured investor rooted in the principles of value investing for over a decade as a fund manager and analyst in the Asian capital markets who started his career at a boutique hedge fund in Singapore where he was with the firm since 2002 and was also part of the core investment committee in significantly outperforming the index in the 10-year-plus-old flagship Asian fund. He was also the portfolio manager for Asia-Pacific equities at Korea’s largest mutual fund company. Prior to setting up the H.E.R.O. Innovators Fund, KB was the Chief Investment Officer & CEO of a Singapore Registered Fund Management Company (RFMC) where he is responsible for listed Asian equity investments. KB had taught accounting at the Singapore Management University (SMU) as a faculty member and also pioneered the 15-week course on Accounting Fraud in Asia as an official module at SMU. KB remains grateful and honored to be invited by Singapore’s financial regulator Monetary Authority of Singapore (MAS) to present to their top management team about implementing a world’s first fact-based forward-looking fraud detection framework to bring about benefits for the capital markets in Singapore and for the public and investment community. KB also served the community in sharing his insights in writing articles about value investing and corporate governance in the media that include Business Times, Straits Times, Jakarta Post, Manual of Ideas, Investopedia, TedXWallStreet. He had also presented in top investment, banking and finance conferences in America, Italy, Sydney, Cape Town, HK, China. He has trained CEOs, entrepreneurs, CFOs, management executives in business strategy & business model innovation in Singapore, HK and China.

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