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1.
JAMA ; 331(14): 1227-1228, 2024 04 09.
Artigo em Inglês | MEDLINE | ID: mdl-38466298

RESUMO

This JAMA Insights discusses the signs and symptoms, diagnosis, and treatment of myotonic dystrophy type 1.


Assuntos
Distrofia Miotônica , Humanos , Mutação , Distrofia Miotônica/classificação , Distrofia Miotônica/diagnóstico , Distrofia Miotônica/genética , Distrofia Miotônica/terapia
2.
Cancer Cell ; 34(2): 331-345.e11, 2018 08 13.
Artigo em Inglês | MEDLINE | ID: mdl-30107179

RESUMO

Therapeutic antibodies targeting ovarian cancer (OvCa)-enriched receptors have largely been disappointing due to limited tumor-specific antibody-dependent cellular cytotoxicity. Here we report a symbiotic approach that is highly selective and superior compared with investigational clinical antibodies. This bispecific-anchored cytotoxicity activator antibody is rationally designed to instigate "cis" and "trans" cytotoxicity by combining specificities against folate receptor alpha-1 (FOLR1) and death receptor 5 (DR5). Whereas the in vivo agonist DR5 signaling requires FcγRIIB interaction, the FOLR1 anchor functions as a primary clustering point to retain and maintain a high level of tumor-specific apoptosis. The presented proof of concept study strategically makes use of a tumor cell-enriched anchor receptor for agonist death receptor targeting to potentially generate a clinically viable strategy for OvCa.


Assuntos
Anticorpos Biespecíficos/uso terapêutico , Receptor 1 de Folato/antagonistas & inibidores , Neoplasias Ovarianas/tratamento farmacológico , Receptores do Ligante Indutor de Apoptose Relacionado a TNF/antagonistas & inibidores , Animais , Linhagem Celular Tumoral , Feminino , Humanos , Masculino , Camundongos , Camundongos Endogâmicos C57BL , Neoplasias Ovarianas/patologia , Receptores de IgG/fisiologia
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