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

Gain of toxic function by long-term AAV9-mediated SMN overexpression in the sensorimotor circuit

长期AAV9介导的SMN在感觉运动回路中的过表达可获得毒性功能。

Van Alstyne, Meaghan; Tattoli, Ivan; Delestrée, Nicolas; Recinos, Yocelyn; Workman, Eileen; Shihabuddin, Lamya S; Zhang, Chaolin; Mentis, George Z; Pellizzoni, Livio

Glucosylceramide in cerebrospinal fluid of patients with GBA-associated and idiopathic Parkinson's disease enrolled in PPMI

PPMI研究中GBA相关性帕金森病和特发性帕金森病患者脑脊液中葡萄糖基神经酰胺的含量

Huh, Young Eun; Park, Hyejung; Chiang, Ming Sum Ruby; Tuncali, Idil; Liu, Ganqiang; Locascio, Joseph J; Shirvan, Julia; Hutten, Samantha J; Rotunno, Melissa S; Viel, Catherine; Shihabuddin, Lamya S; Wang, Bing; Sardi, Sergio Pablo; Scherzer, Clemens R

Dysregulation of Mdm2 and Mdm4 alternative splicing underlies motor neuron death in spinal muscular atrophy

Mdm2和Mdm4选择性剪接失调是脊髓性肌萎缩症运动神经元死亡的根本原因

Van Alstyne, Meaghan; Simon, Christian M; Sardi, S Pablo; Shihabuddin, Lamya S; Mentis, George Z; Pellizzoni, Livio

New Frontiers in Parkinson's Disease: From Genetics to the Clinic

帕金森病研究新前沿:从遗传学到临床

Shihabuddin, Lamya S; Brundin, Patrik; Greenamyre, J Timothy; Stephenson, Diane; Sardi, S Pablo

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

Silencing mutant huntingtin by adeno-associated virus-mediated RNA interference ameliorates disease manifestations in the YAC128 mouse model of Huntington's disease

利用腺相关病毒介导的RNA干扰沉默突变亨廷顿蛋白可改善亨廷顿病YAC128小鼠模型的疾病症状。

Stanek, Lisa M; Sardi, Sergio P; Mastis, Bryan; Richards, Amy R; Treleaven, Christopher M; Taksir, Tatyana; Misra, Kuma; Cheng, Seng H; Shihabuddin, Lamya S

Sustained therapeutic reversal of Huntington's disease by transient repression of huntingtin synthesis

通过暂时抑制亨廷顿蛋白合成,实现亨廷顿病的持续治疗性逆转。

Kordasiewicz, Holly B; Stanek, Lisa M; Wancewicz, Edward V; Mazur, Curt; McAlonis, Melissa M; Pytel, Kimberly A; Artates, Jonathan W; Weiss, Andreas; Cheng, Seng H; Shihabuddin, Lamya S; Hung, Gene; Bennett, C Frank; Cleveland, Don W

Gene transfer to the CNS is efficacious in immune-primed mice harboring physiologically relevant titers of anti-AAV antibodies

在体内含有生理相关滴度抗AAV抗体的免疫预激活小鼠中,基因转移到中枢神经系统是有效的。

Treleaven, Christopher M; Tamsett, Thomas J; Bu, Jie; Fidler, Jonathan A; Sardi, S Pablo; Hurlbut, Gregory D; Woodworth, Lisa A; Cheng, Seng H; Passini, Marco A; Shihabuddin, Lamya S; Dodge, James C

Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy

将反义寡核苷酸递送至小鼠中枢神经系统可缓解严重脊髓性肌萎缩症的症状。

Passini, Marco A; Bu, Jie; Richards, Amy M; Kinnecom, Cathrine; Sardi, S Pablo; Stanek, Lisa M; Hua, Yimin; Rigo, Frank; Matson, John; Hung, Gene; Kaye, Edward M; Shihabuddin, Lamya S; Krainer, Adrian R; Bennett, C Frank; Cheng, Seng H

Neural stem cell transplantation as a therapeutic approach for treating lysosomal storage diseases

神经干细胞移植作为治疗溶酶体贮积症的一种治疗方法

Shihabuddin, Lamya S; Cheng, Seng H