Targeting therapy to the neuromuscular junction: proof of concept.
Targeting therapy to the neuromuscular junction: proof of concept.
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DOI:
10.1002/mus.24057
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发表时间:
2014-05
期刊:
影响因子:
3.4
通讯作者:
Kaminski, Henry J.
中科院分区:
文献类型:
--
作者:
Kusner, Linda L.;Satija, Namita;Cheng, Georgiana;Kaminski, Henry J.
The site of pathology in myasthenia gravis (MG) is the neuromuscular junction (NMJ). Our goal was to determine the ability to direct complement inhibition to the NMJ. Methods: A single-chain antibody directed against the alpha subunit of the acetylcholine receptor was synthesized (scFv-35) and coupled to decay-accelerating factor (DAF, scFv-35-DAF). scFv-35-DAF was tested in a passive model of experimentally acquired MG. Results: Administration of scFv-35-DAF to mice deficient in intrinsic complement inhibitors produced no weakness despite confirmation of its localization to the NMJ and no evidence of tissue destruction related to complement activation. Rats with experimentally acquired MG treated with scFV-35-DAF showed less weakness and a reduction of complement deposition. We demonstrate a method to effectively target a therapeutic agent to the NMJ. Muscle Nerve
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DOI:
10.1073/pnas.80.13.4089
发表时间:
1983-01-01
期刊:
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA-BIOLOGICAL SCIENCES
影响因子:
--
作者:
GOMEZ, CM;RICHMAN, DP
通讯作者:
RICHMAN, DP
影响因子:
64.8
作者:
BEVAN, S;KULLBERG, RW;HEINEMANN, SF
通讯作者:
HEINEMANN, SF
DOI:
10.1073/pnas.75.7.3422
发表时间:
1978-01-01
影响因子:
11.1
作者:
DRACHMAN, DB;ANGUS, CW;KAO, I
通讯作者:
KAO, I
DOI:
10.1073/pnas.84.7.2007
发表时间:
1987-04-01
影响因子:
11.1
作者:
MEDOF, ME;LUBLIN, DM;TYKOCINSKI, ML
通讯作者:
TYKOCINSKI, ML
影响因子:
64.8
作者:
LENNON, VA;LAMBERT, EH
通讯作者:
LAMBERT, EH