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BL-7040

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TLR9

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TLR9激动剂

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Yissum Research Development Co. Ltd.

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溃疡性结肠炎 [+2]

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项与 Ester Neurosciences Ltd. 相关的文献（医药）

2007-08-14·Neurology1区 · 医学

TREATMENT OF HUMAN MYASTHENIA GRAVIS WITH ORAL ANTISENSE SUPPRESSION OF ACETYLCHOLINESTERASE

1区 · 医学

Article

作者: Shlomowitz, N. ; Sussman, J. D. ; Agus, S. ; Brawer, S. ; Soreq, H. ; Yoseph, O. Ben ; McKee, D. ; Argov, Z.

Antisense oligonucleotides are synthetically manufactured short segments of deoxynucleotide sequences that cause targeted gene transcription inhibition. They are designed to interact with their specific complementary mRNA, thereby interfering with transcript stability and/or translation.1,2 This process activates endogenous RNAse-H, which destroys the target mRNA and releases the antisense molecule to act on another mRNA transcript. EN101 antisense (Monarsen) acts against both the catalytic and noncatalytic contributions of acetylcholinesterase (AChE). It was designed to induce destruction of the read-through splicing variant of AChE3 and has shown promising short- and long-term therapeutic effects in experimental myasthenia gravis (MG), both IV and orally.4The objectives of this open label human study with oral EN101 were 1) to evaluate the safety of short-term administration in seropositive MG and 2) to assess possible efficacy by monitoring changes in patients’ functional status. ### Methods. An open label trial was conducted in 16 patients with stable MG receiving at least 180 mg of pyridostigmine daily (for more details see the Neurology Web site at www.neurology.org). For assessment of MG status, the Quantitative MG (QMG) score …

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100 项与 Ester Neurosciences Ltd. 相关的转化医学

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