进行性肌营养不良研究又有新的发现

 肌营养不良研究又有新的发现----干细胞的发现

新干细胞研究,发表在11月科学杂志--自然, 肌营养不良研究人员说： "新干细胞研究为萎缩疾病提供有效的治疗有巨大潜在可能"。
意大利队和法国研究人员发现,这种干细胞可以用来缓解杜氏肌营养不良症状.
研究人员现在用狗做实验，好与人类疾病（肌肉萎缩）症状一一对应. 他们进行了两次试验:
1. 他们从患杜氏肌营养不良狗体内分离出干细胞，添加有益于有遗传缺陷的健康基因，并且把这些细胞注射回这只狗体内. 用这种办法,因为他们想验证自身细胞会不会产生免疫排斥反应。
2. 他们同样从健康的狗体内分离出干细胞, 添加有益于有遗传缺陷的健康基因，并且把这些细胞注射回这只狗体内（患Duchenne）. 用这种办法,因为他们想验证自身细胞会不会产生免疫排斥反应。
总的来说,注射干细胞取自于健康犬获得很大改善. 四狗分别接受捐赠者的健康干细胞，其中早期两只狗能够正常走路和另两只早期死亡. 其中后者可能没有足够的干细胞注射. 研究者还用健康的干细胞注射到在后一阶段患疾病老年狗的体内,这些狗存活的时间比他们预期时间要长。
虽然这些成果很令人兴奋,但是需要做更多的研究调查工作,来验证这种方法是否会对人体有效. 还有一个问题是研究者怎样培养出足够的人体干细胞来治疗Duchenne疾病患者; 第二个问题是这种治疗是否对骨骼肌、心脏肌肉也有同样的效果.
肌营养不良的研究主任Dr. Marita Pohlschmidt博士说:
"肌肉萎缩症组织发现干细胞疗法能够治疗修补受损和衰败的肌肉组织.我们很受鼓舞，因为他的同事发表Cossu初步结果证明,为我们用干细胞治疗杜氏肌营养不良向前迈进一步.我们的研究还处于早期阶段,在这一技术可应用于人体之前有许多问题有待解决.更重要的是,若成功的证明此治疗方法在人体同样有效,这一技术有可能发展成为一个不但有效的治疗Duchenne,而且同样能够治疗其他肌肉损伤和疾病."
这项研究详细资料可查阅www.muscular-dystrophy.org
Duchenne肌营养不良
杜氏肌营养不良症是一种遗传造成的营养不良蛋白基因和遗传缺陷是几种肌营养不良. 肌营养不良造成肌肉麻痹肌肉细胞逐渐破裂而死. 杜氏肌营养不良症,是用医生名字命名的， 1800年发现此病,几乎只有男性患者. 目前没有医治或治疗杜氏肌营养不良. 关于干细胞疗法
干细胞疗法的潜在价值早已修补受损肌肉组织或衰败.研究人员利用一种称为mesoangioblast干细胞,可以取自血脉. 这些细胞有能力取代,但他们同时是"突变"变成肌肉细胞. 他们可以在人体外的生长和组织培养可以穿过血管壁肌肉和入侵时回注到血液. 一旦肌肉,可以修复这个惊人的效率. 因此使用这些干细胞是一种潜在的开创性地治疗杜氏肌营养不良.
对进行性肌营养不良
肌肉萎缩症组织每年投入300万英镑支持关怀服务中心肌肉、网络、信息资源和研究. 它摸索寻求治疗方法,并有40多年的实践,医疗及雄厚的医资力量条件.
在英国已经有相关肌肉或肌营养不良症有3万余人,. 另外120,000(老者,父母兄弟姐妹)受遗传影响. 这些条件是最能影响人的遗传、年龄、背景和民族.
http://www.amrc.org.uk/index.asp?id=21757
Muscular Dystrophy Campaign encouraged by new stem cell findings

New stem cell research, published in the November issue of the leading science journal, Nature, "has the potential to develop into an efficient and groundbreaking treatment", according to the Muscular Dystrophy Campaign.
在十一月的领头科学杂志，自然界涌出中出版新干细胞研究有变为生长潜力一有效率和根据肌营养不良运动破天荒的处理
The team of Italian and French researchers working on this found that stem cells can be used to alleviate the symptoms of Duchenne muscular dystrophy.
The researchers used a dog model for Duchenne which is currently the best counterpart to the human disease. They carried out two experiments:
1. They isolated stem cells from dogs with Duchenne muscular dystrophy, added a healthy gene to correct the genetic defect and injected these cells back into the same donor. They investigated this approach because using an individual抯 own cells minimises the chances of rejection and avoids the need for immunosuppression.
2. They isolated stem cells from healthy dogs of the same strain and injected these cells into dogs with Duchenne, while giving the recipients an immunosuppressant.
Overall, the injections of stem cells taken from healthy dogs brought about the most improvement. Of four dogs who received the stem cells from a healthy donor at an early stage, two retained the ability to walk and two died early. One of the latter ones might not have been injected with enough cells. The researchers also injected healthy stem cells into older dogs at a later stage of the disease and these dogs lived longer than expected and had their mobility restored.
Although these results are promising, a lot more research needs to be done to investigate whether this approach to treatment might work in humans. One of the questions researchers still have is whether human stem cells of this type can be grown in sufficient numbers to have a therapeutic benefit when injected into the bloodstream of a boy with Duchenne. A second question to investigate is whether the current approach, which concentrates solely on skeletal muscle, has the same effect on heart muscle.
Director of Research at the Muscular Dystrophy Campaign, Dr. Marita Pohlschmidt, said:
"The Muscular Dystrophy Campaign recognises the potential of stem cell therapy for the repair of damaged and degenerated muscle tissue. We feel encouraged by the work published by Cossu and his colleagues because the results provide initial evidence that we might be one step closer to a stem cell treatment for Duchenne muscular dystrophy. The research is still at an early stage and there are a number of issues to be resolved before this technology can be tested on humans. Importantly though, if it does prove to be successful in humans, this technology has the potential to develop into an efficient and groundbreaking treatment not only for Duchenne, but also other muscular dystrophies."
Further information on this research can be found on www.muscular-dystrophy.org
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy is a genetic disorder caused by an error in the dystrophin gene and is one of several types of muscular dystrophy. Muscular dystrophy causes progressive muscle weakness as muscle cells break down and die. Duchenne muscular dystrophy, named after the doctor who first studied this specific condition in the 1800s, affects almost exclusively boys. There are currently no treatments or cures for Duchenne muscular dystrophy.
About Stem Cell Therapy
Stem cell therapy has long been valued for its potential to repair damaged or degenerated muscle tissue. In the work reported here, researchers used a type of stem cell called a mesoangioblast, which can be harvested from blood vessels. These cells have the ability to replace themselves, but they are also "programmed" to turn into muscle cells. They can be grown outside the body in tissue culture and can pass through the blood vessel walls and invade the muscle when they are injected back into the bloodstream. Once they are in muscle, they can repair this with surprisingly high efficiency. The use of these stem cells therefore represents a potentially groundbreaking way to develop a treatment for Duchenne muscular dystrophy.
About the Muscular Dystrophy Campaign
Muscular Dystrophy Campaign invests ? million a year in care support services, muscle centres, networks, information, resources and research. It has pioneered the search for treatments and cures for over 40 years and provides practical, medical and emotional support to people affected by the condition.
More than 30,000 people in the UK have muscular dystrophy or a related muscle disease. A further 120,000 (carers, parents, siblings) are affected. Most of these conditions are genetic and can affect people of all ages, backgrounds and nationalities.

 

转载于:https://www.cnblogs.com/Gemgin/archive/2013/06/13/3136601.html