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

Hematopoietic Stem-Cell Gene Therapy for Cystinosis

胱氨酸病的造血干细胞基因治疗

Barshop, Bruce A; Ball, Edward D; Benador, Nadine; Trauner, Doris; Phillips, Susan; Dohil, Ranjan; Afshari, Natalie A; Roy, Sohini; Campo Fernandes, Beatriz; Kohn, Donald; Shayan, Katayoon; Everett, John K; Bushman, Frederic D; Midgley, Julian; Liang, Hong; Sawyers, Anne; Gangoiti, Jon A; Panchal, Maulik; Ahmed, Imama; Cherqui, Stephanie

Contaminating plasmid sequences and disrupted vector genomes in the liver following adeno-associated virus gene therapy

腺相关病毒基因治疗后肝脏中存在的污染质粒序列和破坏的载体基因组

Buddle, Sarah; Brown, Li-An K; Morfopoulou, Sofia; Torres Montaguth, Oscar Enrique; Scoto, Mariacristina; Herder, Vanessa; Dhawan, Anil; Brown, Julianne R; Atkinson, Laura; Kopec, Angelika; Davis, Dee; Storey, Nathaniel; Campos, Luis; Sebire, Neil; Macpherson, Hannah; Lee, Jasmaine; Orton, Richard; Baranello, Giovanni; Asamaphan, Patawee; Ilia, Georgios; Karda, Rajvinder; Belfield, Holly; Counsell, John; Waddington, Simon N; Thomson, Emma C; Muntoni, Francesco; Breuer, Judith

Gene Therapy-Related Malignancy and the Risk of Cancer Exceptionalism

基因治疗相关恶性肿瘤与癌症特殊性风险

Benedetti, Daniel J; Marron, Jonathan M; Kodish, Eric

Engineered coagulation factor VIII with enhanced secretion and coagulation potential for hemophilia A gene therapy

用于血友病A基因治疗的具有增强分泌和凝血潜能的工程化凝血因子VIII

Kashiwakura, Yuji; Nakajima, Yuto; Horinaka, Kio; Lopes, Tiago J S; Furuta, Yuma; Yamaguchi, Yuki; Baatartsogt, Nemekhbayar; Hayakawa, Morisada; Katakai, Yuko; Uchiyama, Susumu; Nureki, Osamu; Nogami, Keiji; Ohmori, Tsukasa

Out-of-Pocket Cost of Spinal Muscular Atrophy Gene Therapies Among Commercially Insured Patients

商业保险患者自付脊髓性肌萎缩症基因治疗费用

Sckaff, Maria; Odouard, Ilina C; Ballreich, Jeromie; Anderson, Gerard; Socal, Mariana P

Postnatal Slc26a4 gene therapy improves hearing and structural integrity in a hereditary hearing loss model

产后Slc26a4基因治疗可改善遗传性听力损失模型中的听力和结构完整性

Tsai, Yi-Hsiu; Wu, Peng-Yu; Chuang, Yu-Chi; Huang, Chun-Ying; Takeda, Hiroki; Hibino, Hiroshi; Wu, Chen-Chi; Cheng, Yen-Fu

Gene therapy via CRISPR/Cas9-mediated Cxcr4 disease allele inactivation reverses leukopenia in WHIM mice

利用 CRISPR/Cas9 介导的 Cxcr4 致病等位基因失活进行基因治疗可逆转 WHIM 小鼠的白细胞减少症。

Gao, Ji-Liang; Li, Zhanzhuo; Calderon-Perez, Rafael; Pavek, Antonia; Kim, Lina; McDermott, David H; Murphy, Philip M

AAV-mediated gene therapy in a model of SLC13A5 citrate transporter disorder rescues epileptic and metabolic phenotypes.

在 SLC13A5 柠檬酸转运蛋白疾病模型中,AAV 介导的基因治疗可挽救癫痫和代谢表型。

Bailey Lauren E, Adams Raegan M, Schackmuth Morgan K, Garza Irvin T, Knight Krishanna, Holmes Sydni K, Eller Meghan M, Lee MinJae, Bailey Rachel M

Correction: Transdermal delivery of CRISPR/Cas9-mediated melanoma gene therapy via polyamines-modified thermosensitive hydrogels

更正:通过聚胺修饰的热敏水凝胶进行CRISPR/Cas9介导的黑色素瘤基因治疗的透皮递送

Li, Meng; Zhou, Songli; Zhang, Suqin; Xie, Xingyu; Nie, Junqi; Wang, Qi; Ma, Lixin; Cheng, Yibin; Luo, Jingwen

AAV9 gene therapy optimization for SMARD1/CMT2S: safety and long-term efficacy comparison of two vectors in a SMARD1 preclinical model

针对SMARD1/CMT2S的AAV9基因治疗优化:在SMARD1临床前模型中比较两种载体的安全性和长期疗效

Pagliari, Elisa; Anastasia, Alessia; Bellandi, Floriana; Garbellini, Manuela; Ongaro, Jessica; Taiana, Michela; Comi, Giacomo P; Ottoboni, Linda; Sierra-Delgado, Julieth Andrea; Likhite, Shibi; Meyer, Kathrin C; Nizzardo, Monica; Corti, Stefania P