V***b 发帖数: 3419 | 1 比如SMA (Spinal Muscular Atrophy) 这种疾病是由于一个叫SMN1的隐性基因缺失引起
的,父母都没有病症但是都缺一个allele,结果孩子很不幸,25%的可能性发生了,
SMN1编码的蛋白SMN没有表达,这个蛋白是负责mRNA剪切的,对运动神经元的存活至关
重要,结果孩子终生不能走和运动,以后吃饭呼吸还会有困难。这种一个基因完全没有
的情况用病毒做载体感染可行吗?肯定有人试过了。主要的难关在哪里?转染效率低?
特定细胞表面没有受体?基因整合困难? |
n********k 发帖数: 2818 | 2 If I don't remember wrong, it is in clincial phase or at least close, one of
the hottest/competitive area and tons of people are thinking about it,
myself included but would be for something different and a next generation
approach too...I am pretty optimistic about this and I believe some studies
along the similar lines are at the point of some breakthrough could happen
anytime within next 3 to 5ys...
【在 V***b 的大作中提到】![](/moin_static193/solenoid/img/up.png) : 比如SMA (Spinal Muscular Atrophy) 这种疾病是由于一个叫SMN1的隐性基因缺失引起 : 的,父母都没有病症但是都缺一个allele,结果孩子很不幸,25%的可能性发生了, : SMN1编码的蛋白SMN没有表达,这个蛋白是负责mRNA剪切的,对运动神经元的存活至关 : 重要,结果孩子终生不能走和运动,以后吃饭呼吸还会有困难。这种一个基因完全没有 : 的情况用病毒做载体感染可行吗?肯定有人试过了。主要的难关在哪里?转染效率低? : 特定细胞表面没有受体?基因整合困难?
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b*****o 发帖数: 150 | 3 我觉得还是安全性和有效性吧
病毒的gene therapy已经死过人了
而且即使安全 怎么才能有效感染神经呢?
【在 V***b 的大作中提到】![](/moin_static193/solenoid/img/up.png) : 比如SMA (Spinal Muscular Atrophy) 这种疾病是由于一个叫SMN1的隐性基因缺失引起 : 的,父母都没有病症但是都缺一个allele,结果孩子很不幸,25%的可能性发生了, : SMN1编码的蛋白SMN没有表达,这个蛋白是负责mRNA剪切的,对运动神经元的存活至关 : 重要,结果孩子终生不能走和运动,以后吃饭呼吸还会有困难。这种一个基因完全没有 : 的情况用病毒做载体感染可行吗?肯定有人试过了。主要的难关在哪里?转染效率低? : 特定细胞表面没有受体?基因整合困难?
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n********k 发帖数: 2818 | 4 ok here are the two groups leading the field:
Brian Kasper in USA and
Mimoun Azzouz in UK
We are developing some potential collaborations with one of the two groups
and I am trying to get connected with a second one...but what I am planning
on are quite different from them, but we need their expertise too.
Frankly this is exactly the reason why some of us shall go back, simply
there are a lot more opportunity to do some beautiful work at the very
cutting edge and compete at a different level...I wouldn't even think to do
any of these if I had chosen to stay in the states. Just heard from a long-
lost good buddy who was behind a famous website and went back to China last
year...
【在 b*****o 的大作中提到】![](/moin_static193/solenoid/img/up.png) : 我觉得还是安全性和有效性吧 : 病毒的gene therapy已经死过人了 : 而且即使安全 怎么才能有效感染神经呢?
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b*****o 发帖数: 150 | 5 aav 是个相对promising 安全的载体 和retro比起来
但不太明白他那个是怎么进入神经的 明天到单位再看他的paper
题外话 老夫当年宿舍卧谈的时候还臆想那改造的rabies病毒去做基因治疗的载体 对付
神经系统疾病 现在想来真是 哈哈哈哈 |
u**********d 发帖数: 573 | 6 对,靶向性应该是影响有效性的一个重要因素。
【在 b*****o 的大作中提到】![](/moin_static193/solenoid/img/up.png) : 我觉得还是安全性和有效性吧 : 病毒的gene therapy已经死过人了 : 而且即使安全 怎么才能有效感染神经呢?
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V***b 发帖数: 3419 | 7 管他什么靶向不靶向的,全身细胞都被感染不就得了。估计还是感染效率和基因整合效
率低?我是看到这个才思考了一下,可惜不是这个方向的。http://www.mitbbs.com/article_t/NextGeneration/35184945.html
【在 u**********d 的大作中提到】![](/moin_static193/solenoid/img/up.png) : 对,靶向性应该是影响有效性的一个重要因素。
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r****r 发帖数: 36 | 8 gene therapy using viral vector is just like doing RANDOM MUTAGENESIS in
human body. |
n********k 发帖数: 2818 | 9 alright, I would rarely recommend anything medically unless I am very sure..
.however, after reading this very unfortunate but very touching story, I
would recommend they contact People like Dr. Brian Kasper in Ohio(I
personally know him); and he has been in the field for almost 20 ys now. The
AAV gene therapy approach he has been testing first in mice and now in
primate seems very effective and likely safe to me. Sure, it is not yet
clinically tested but I think this is a case very much justified to take
some risk...The girl is only three now and it is also better/easier before
too much damages are done due to the long-term denervation
【在 V***b 的大作中提到】![](/moin_static193/solenoid/img/up.png) : 管他什么靶向不靶向的,全身细胞都被感染不就得了。估计还是感染效率和基因整合效 : 率低?我是看到这个才思考了一下,可惜不是这个方向的。http://www.mitbbs.com/article_t/NextGeneration/35184945.html
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n********k 发帖数: 2818 | 10 AAV9 infection efficiency either through spinal cord or blood is not that
bad and it doesn't need integration either. SMA would be one of the easiest
targets for gene therapy...
【在 V***b 的大作中提到】![](/moin_static193/solenoid/img/up.png) : 管他什么靶向不靶向的,全身细胞都被感染不就得了。估计还是感染效率和基因整合效 : 率低?我是看到这个才思考了一下,可惜不是这个方向的。http://www.mitbbs.com/article_t/NextGeneration/35184945.html
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a*****u 发帖数: 493 | |