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

Efficient in vivo mammalian neuron editing using peptide-mediated CRISPR enzyme delivery

利用肽介导的CRISPR酶递送实现高效的体内哺乳动物神经元编辑

Manohar, Brigette D; Travis, Meika; Munjal, Vikas; Baehr, Christopher M; Rahnama, Lily M A; Kang, Min Hyung; Haddad, Rania F; Asija, Kunica; Noel, Eric A; Peddireddy, Navya; Chouhan, Runvir S; Sharma, Rohit; Wyman, Stacia K; Krishnappa, Netravathi; Killebrew, Deirdre A; Li, Song Hua; Snow, Kathy J; Byrne, Addison K; Boosani, Chandra S; Otero, P Anthony; Bringas, John; O'Brien, Allison A; Rocco, Matthew T; Zimmerly, Madison P; Gray, Isabelle; Sran, Gunita K; Patel, Mia I; Seidensticker, Emily F; Saville, Ethan; Gaitan, Yaned; Schmelzle, Amanda L; Subramanyam, Pradeep Nag B; Samaranch, Lluis; Green, Jonathan A; Wells, Kevin D; Ehrenberg, Alexander J; Murray, Stephen A; Clelland, Claire D; Murthy, Niren; Lonser, Russell R; Hadaczek, Piotr; Van Laar, Victor S; Bankiewicz, Krystof S; Wilson, Ross C

Persistent GDNF Expression 45 Months after Putaminal Infusion of AAV2-GDNF in a Patient with Parkinson's Disease

帕金森病患者壳核内注射AAV2-GDNF 45个月后,GDNF表达持续存在

Heiss, John D; Ray-Chaudhury, Abhik; Kleiner, David E; Ehrlich, Debra J; Scott, Gretchen; Edwards, Nancy A; Goldstein, David S; Hammoud, Dima A; Hadaczek, Piotr; Van Laar, Victor S; Graff, Shantelle A; Herscovitch, Peter; Lungu, Codrin; Hallett, Mark; Lonser, Russell R; Zaghloul, Kareem A; Bankiewicz, Krystof S

GDNF gene therapy for alcohol use disorder in male non-human primates

GDNF 基因治疗雄性非人类灵长类动物酒精使用障碍

Matthew M Ford #, Brianna E George #, Victor S Van Laar #, Katherine M Holleran #, Jerusha Naidoo, Piotr Hadaczek, Lauren E Vanderhooft, Emily G Peck, Monica H Dawes, Kousaku Ohno, John Bringas, Jodi L McBride, Lluis Samaranch, John R Forsayeth, Sara R Jones, Kathleen A Grant, Krystof S Bankiewicz

Adeno-associated viral vector serotype 9-based gene therapy for Niemann-Pick disease type A

基于腺相关病毒载体9型血清型的A型尼曼匹克病基因治疗

Lluis Samaranch, Azucena Pérez-Cañamás, Beatriz Soto-Huelin, Vivek Sudhakar, Jerónimo Jurado-Arjona, Piotr Hadaczek, Jesús Ávila, John R Bringas, Josefina Casas, Haifeng Chen, Xingxuan He, Edward H Schuchman, Seng H Cheng, John Forsayeth, Krystof S Bankiewicz, María Dolores Ledesma

Kinetics and MR-Based Monitoring of AAV9 Vector Delivery into Cerebrospinal Fluid of Nonhuman Primates

非人灵长类动物脑脊液中AAV9载体递送的动力学和基于磁共振的监测

Ohno, Kousaku; Samaranch, Lluis; Hadaczek, Piotr; Bringas, John R; Allen, Philip C; Sudhakar, Vivek; Stockinger, Diane E; Snieckus, Christopher; Campagna, Michael V; San Sebastian, Waldy; Naidoo, Jerusha; Chen, Haifeng; Forsayeth, John; Salegio, Ernesto A; Hwa, Granger G C; Bankiewicz, Krystof S

Extensive Transduction and Enhanced Spread of a Modified AAV2 Capsid in the Non-human Primate CNS

改良的 AAV2 衣壳在非人类灵长类动物中枢神经系统中具有广泛的转导和增强的扩散

Jerusha Naidoo, Lisa M Stanek, Kousaku Ohno, Savanah Trewman, Lluis Samaranch, Piotr Hadaczek, Catherine O'Riordan, Jennifer Sullivan, Waldy San Sebastian, John R Bringas, Christopher Snieckus, Amin Mahmoodi, Amir Mahmoodi, John Forsayeth, Krystof S Bankiewicz, Lamya S Shihabuddin

Widespread AAV1- and AAV2-mediated transgene expression in the nonhuman primate brain: implications for Huntington's disease

非人灵长类动物大脑中广泛的AAV1和AAV2介导的转基因表达:对亨廷顿病的启示

Hadaczek, Piotr; Stanek, Lisa; Ciesielska, Agnieszka; Sudhakar, Vivek; Samaranch, Lluis; Pivirotto, Philip; Bringas, John; O'Riordan, Catherine; Mastis, Bryan; San Sebastian, Waldy; Forsayeth, John; Cheng, Seng H; Bankiewicz, Krystof S; Shihabuddin, Lamya S

Distribution of nanoparticles throughout the cerebral cortex of rodents and non-human primates: Implications for gene and drug therapy

纳米颗粒在啮齿动物和非人灵长类动物大脑皮层的分布:对基因和药物治疗的启示

Salegio, Ernesto A; Streeter, Hillary; Dube, Nikhil; Hadaczek, Piotr; Samaranch, Lluis; Kells, Adrian P; San Sebastian, Waldy; Zhai, Yuying; Bringas, John; Xu, Ting; Forsayeth, John; Bankiewicz, Krystof S

SAFETY AND TOLERABILITY OF MRI-GUIDED INFUSION OF AAV2-hAADC INTO THE MID-BRAIN OF NON-HUMAN PRIMATE

MRI 引导下将 AAV2-hAADC 输注到非人类灵长类动物中脑的安全性和耐受性

Waldy San Sebastian, Adrian P Kells, John Bringas, Lluis Samaranch, Piotr Hadaczek, Agnieszka Ciesielska, Michael Macayan, Phillip J Pivirotto, John Forsayeth, Sheryl Osborne, J Fraser Wright, Foad Green, Gregory Heller, Krystof S Bankiewicz

Cerebral infusion of AAV9 vector-encoding non-self proteins can elicit cell-mediated immune responses.

将编码非自身蛋白的 AAV9 载体注入大脑可以引发细胞介导的免疫反应

Ciesielska Agnieszka, Hadaczek Piotr, Mittermeyer Gabriele, Zhou Shangzhen, Wright J Fraser, Bankiewicz Krystof S, Forsayeth John