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武田与UCL合作开发基因疗法和干细胞疗法 [复制链接]

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楼主
发表于 2014-3-13 16:03 |只看该作者 |倒序浏览 |打印
作者:tomato 来源:生物谷
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2014年3月11日讯 /生物谷BIOON/ --武田(Takeda)3月10日宣布,与伦敦大学学院(UCL)达成合作,推动肌肉疾病研究,尤其是肌营养不良症(muscular dystrophy)。该项研究将由UCL的Francesco团队领导,武田的新领域科学研究组(NFS group)提供资金支持。NFS的目的是支持可能导致新药发现和开发的创新、尖端科学研究。  e! C- T  t0 W5 \8 Q7 ~
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Tedesco博士领导的团队一直专注于肌肉再生研究、以及干细胞疗法用于肌营养不良症治疗潜力的研究,尤其是诱导多能干细胞(iPS)。该团队也正在开展相关研究,利用人工染色体和新型生物材料开发新颖基因疗法和细胞治疗策略,用于肌营养不良症治疗。/ E/ H: V  W/ N* G) w4 z, b5 B' a
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通过此前在杜氏肌营养不良症小鼠模型中的研究工作,该团队已证明了一种人工合成染色体基因替代疗法的临床潜力。此外,通过将iPS细胞移植入另一种遗传性肌病(肢带肌营养不良症2D)小鼠模型的研究,Tedesco团队也证明了遗传矫正的iPS细胞的临床潜力9 }7 C! }: @- s  x% j3 S
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发表于 2014-3-13 16:03 |只看该作者
Takeda and UCL to work together to tackle muscle disorders3 \+ U5 z4 j. w' [/ D
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Japanese pharmaceutical company Takeda will work with University College London (UCL) to drive research into tackling muscle disorders, in particular muscular dystrophy.9 ~( s. F8 A' \- M- D8 v

; H% g( U: x% e6 I. x1 \; W/ sThe research – which is being conducted by the research group of Dr Francesco Saverio Tedesco – is being supported through funding of $250,000 from the company's New Frontier Sciences group. Takeda's NFS aims to support innovative, cutting-edge research which could eventually lead to drug discovery and development.
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Dr Tedesco's team will focus on the study of muscular regeneration and the potential for stem cell therapies to treat muscular dystrophy, in particular induced pluripotent (iPS) stem cells.
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) ?( ^6 G9 {# X  ~The team is also investigating the potential for treating muscular dystrophy through developing novel gene and cell therapy strategies using artificial human chromosomes and novel biomaterials.
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6 Y' M3 n/ o! H( m7 O2 H4 XUsing this approach, Dr Tedesco hopes to overcome a number of current limitations to developing effective treatments for muscular dystrophies. It is hoped that through the use of these modified stem cells, large quantities of progenitor cells could be produced to be transplanted into a patient's muscle following genetic correction or to be used for drug development platforms.( I8 I9 {4 ]9 q5 W  t2 L

% S, K$ u8 s+ s& d* SImportantly, the team will attempt to produce these cells which can be applied more easily in a clinical context, in order to reduce the hurdles that might limit their possible future use in clinical studies.
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Through previous work using a mouse model of Duchenne muscular dystrophy, the team has already demonstrated the potential of pre-clinical gene replacement therapy using an artificial human chromosome.! i: x' _6 t; A' c* u

5 N9 D) b, \# ]4 R3 G7 aMoreover, in a separate study, Dr Tedesco and his team also demonstrated the potential of genetically corrected iPS cells which had been transplanted into another mouse model of a genetic muscle disorder (limb-girdle muscular dystrophy 2D).
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Commenting on the new funding for his research, Dr Tedesco, from the UCL Department of Cell and Developmental Biology, said:/ _+ _$ B) K+ J5 A; w
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"This funding from Takeda comes at a crucial time for building on our research to develop new and innovative approaches to developing potentially life-changing treatments for muscular dystrophies.1 j/ [; T5 C4 E1 P1 F

! z2 K( g0 Q/ Q"With Takeda's backing, my team and I will be building on the processes we've already shown to be promising and which could pave the way for the development of novel strategies for both cell therapies and drug discovery in muscle disorders."
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9 [+ T, ?7 A; z" k! }Gordon Wong, D.Phil., Head of New Frontier Science at Takeda, added:5 S) I; I4 k( B7 I5 d* a) j
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"We are delighted to support Dr Tedesco and his team because their ground breaking work has the potential for significant patient benefit."
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"That several different strands of their research have already borne fruit was strong evidence for us of the translational potential of Dr Tedesco's research for muscular dystrophies."5 H6 x4 v+ q5 S: ^3 f* \
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