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【bio-news】Duchenne型肌营养不良症有望治疗:胚胎

Embryonic Stem Cells Create Healthy Muscle in Mice

SUNDAY, Jan. 20 (HealthDay News) -- Researchers have coaxed embryonic mouse stem cells to grow into healthy muscle tissue, in a feat that creates new possibilities for the treatment of Duchenne muscular dystrophy (DMD).

DMD is the most common of nine types of muscular dystrophy, which is characterized by a lack of the protein dystrophin in voluntary muscles, such as those in the arms and legs. Dystrophin plays a key role in building and repairing muscle; without it, muscles deteriorate and lose function.

The University of Texas Southwestern Medical Center team focused on developing embryonic stem cells containing the gene Pax3, which triggers cells to grow into muscle tissue that will produce dystrophin.

"Embryonic stem cells can make every tissue in the body. We instructed these cells to make more skeletal muscle, and from a crowd of cells," explained study author Rita Perlingeiro. "We found a way to pull out only the ones destined to make muscle. These two steps combined resulted in a cell population capable of making muscle in a mouse with muscular dystrophy and, very importantly, the new muscle is stronger."

This is one of the few studies to test the ability of embryonic stem cells to grow in adult muscle tissue, the researchers added. The method they used also managed to avoid the risk of tumor formation in the mice.

One expert lauded the study, which appears in the Jan. 20 online issue ofNature Medicine, as a strong first step.

"By way of experiments done with mice, the paper offers a compelling 'proof of principle,' that embryonic stem cells can be turned into muscle-producing cells in the laboratory and used to deliver healthy muscle to people with Duchenne muscular dystrophy," said Paul Muhlrad, research program coordinator for the Muscular Dystrophy Association.

The researchers noted it was only necessary to regenerate a portion of the muscle tissue for the mice to regain some control. However, the process requires refining before it can be tried in humans, they added.

"At the present time, no one has yet demonstrated that genetic manipulation of human embryonic stem cells can be used to derive functional skeletal muscle progenitors from these cells, so it's far too early to tell whether this technique could lead to any potential clinical application," said Perlingeiro. "The main hurdle is to make sure we can indeed combine successfully these two approaches, and test these cells exhaustively in mouse models before we think about clinical trials."

Muhlrad also cautioned that this research is a long way from human use.

"While mice provide an excellent model system, experiments that work in mice don't always readily transfer to humans. Scientists would probably want to replicate the experiments in dog models of muscular dystrophy before moving on to human studies," Muhlrad said. Additionally, the mice had to take immunosuppressants to prevent their bodies from rejecting cells from another mouse. The ideal approach would be to use a body's own stem cells to avoid the issue of rejection.

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胚胎干细胞能产生健康肌肉组织

SUNDAY, Jan. 20 (HealthDay News) -- Researchers have coaxed embryonic mouse stem cells to grow into healthy muscle tissue, in a feat that creates new possibilities for the treatment of Duchenne muscular dystrophy (DMD).

1月20日,星期日,(HealthDay News)研究人员已经成功将小鼠胚胎干细胞诱导分化为健康的肌肉组织。这一创造性的贡献为进行性假肥大性肌营养不良(DMD)提供了一种新的治疗手段。

DMD is the most common of nine types of muscular dystrophy, which is characterized by a lack of the protein dystrophin in voluntary muscles, such as those in the arms and legs. Dystrophin plays a key role in building and repairing muscle; without it, muscles deteriorate and lose function.

DMD是九种肌营养不良症的最常见类型,以缺少四肢等随意肌的肌营养不良蛋白为主要特征。肌营养不良蛋白在肌肉的生长和修复中扮演了重要的角色,如果没有这种蛋白,肌肉就会变性和失去功能。

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(Science News --编译 )

作者:admin@医学,生命科学    2011-05-05 17:46
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