日期:
2020 年 — 2026 年
2020
2021
2022
2023
2024
2025
2026
影响因子:

FLT201, a novel liver-directed AAV gene therapy candidate for Gaucher disease type 1.

FLT201 是一种新型的肝脏靶向 AAV 基因治疗候选药物,用于治疗 1 型戈谢病。

Comper Fabrizio, Miranda Carlos J, Liou Benjamin, Dodev Tihomir, Jeyakumar Jey M, Canavese Miriam, Cocita Clement, Khoshrou Khashayar, Tiscornia Gustavo, Chisari Elisa, Stotter Emmaline, Shehu Erald, Sridharan Sudharsan, Yu I-Mei, Pandya Jalpa, Khinder Jaminder, Northcott Natalie, Kalcheva Petya, Correia Samantha, Sun Ying, Dane Allison P, Sheridan Rose, Nathwani Amit C, Corbau Romuald

Differences in wild-type- and R338L-tenase complex formation are at the root of R338L-factor IX assay discrepancies

野生型和R338L突变型凝血酶原酶复合物形成差异是R338L突变型凝血因子IX检测结果不一致的根本原因。

Foley, Jonathan H; Shehu, Erald; Riddell, Anne; Gray, Elaine; Goodale, Andrew; Yu, I-Mei; Verhoef, Daniël; Little, John; Shattock, Diane; Kitchen, Steve; Chowdary, Pratima; Corbau, Romuald; Nathwani, Amit C

RNAi prevents and reverses phenotypes induced by mutant human ataxin-1

RNAi可预防和逆转突变型人类共济蛋白-1诱导的表型

Keiser, Megan S; Monteys, Alejandro Mas; Corbau, Romuald; Gonzalez-Alegre, Pedro; Davidson, Beverly L

Lersivirine, a nonnucleoside reverse transcriptase inhibitor with activity against drug-resistant human immunodeficiency virus type 1.

勒西韦林是一种非核苷类逆转录酶抑制剂,对耐药性人类免疫缺陷病毒1型具有活性

Corbau Romuald, Mori Julie, Phillips Chris, Fishburn Lesley, Martin Alex, Mowbray Charles, Panton Wendy, Smith-Burchnell Caroline, Thornberry Adele, Ringrose Heather, Knöchel Thorsten, Irving Steve, Westby Mike, Wood Anthony, Perros Manos