Treatment of Neuronopathic Mucopolysaccharidoses with Blood-Brain Barrier-Crossing Enzymes: Clinical Application of Receptor-Mediated Transcytosis

• Үндсэн зохиолчид: Hiroyuki Sonoda (Зохиогч), Kenichi Takahashi (Зохиогч), Kohtaro Minami (Зохиогч), Toru Hirato (Зохиогч), Tatsuyoshi Yamamoto (Зохиогч), Sairei So (Зохиогч), Kazunori Tanizawa (Зохиогч), Mathias Schmidt (Зохиогч), Yuji Sato (Зохиогч)
• Формат: Ном
• Хэвлэсэн: MDPI AG, 2022-06-01T00:00:00Z.
• Нөхцлүүд: article
• Онлайн хандалт: Connect to this object online.

Enzyme replacement therapy (ERT) has paved the way for treating the somatic symptoms of lysosomal storage diseases (LSDs), but the inability of intravenously administered enzymes to cross the blood-brain barrier (BBB) has left the central nervous system (CNS)-related symptoms of LSDs largely impervi...