CAP-001: Systemic AAV Gene Therapy with Next Generation Capsids for MPS II Disease

被引：0

作者：

Ou, Li ^{[1
]}

Octeau, Christopher ^{[1
]}

Bonet, Bailey ^{[1
]}

Lam, Kevin ^{[1
]}

Siemian, Justin ^{[1
]}

Dreher, Renee ^{[1
]}

Roxas, Kristina ^{[1
]}

Jeffreys, David ^{[1
]}

Thomas, Shelby ^{[1
]}

Grepo, Nicole ^{[1
]}

Sandrock, Robert ^{[1
]}

Cockrell, Adam ^{[1
]}

Scherrer, Jon ^{[1
]}

Ressler, Reed ^{[1
]}

Wheeler, Brandon ^{[1
]}

Badger, Drew ^{[1
]}

Knoll, Allison ^{[1
]}

Goeden, Nick ^{[1
]}

机构：

[1] Capsida Biotherapeut Inc, Thousand Oaks, CA USA

来源：

MOLECULAR THERAPY | 2022年 / 30卷 / 04期

关键词：

D O I：

暂无

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

471

引用

页码：223 / 223

页数：1

共 44 条

[21] Systemic L-DOPA Secretion by AAV Gene Therapy to Improve the Treatment of Parkinson's Disease
Antunes, Andre Saraiva Leao Marcelo
Hikima, Atsuko
Hobbs, Carl
McDonald, Michael
Rose, Sarah
Henckaerts, Els
HUMAN GENE THERAPY, 2016, 27 (07) : A11 - A11
[22] Systemic AAV Gene Therapy with CNSTargeted Engineered Capsids Achieves Significant GCase Activity Increases in the Primate Brain to Support the Potential Treatment of GBA-PD
McDowell, Kimberly
Ressler, Reed
Wheeler, Brandon
Thomas, Shelby
Grigoryeva, Lubov S.
Armendariz, Alexander
Flynn, Maeve
Acharya, Hari
Denis, Paul
Tchourilov, Pasha
Tole, Swati
Goeden, Nick
Flytzanis, Nicholas C.
Catalano, Susan
MOLECULAR THERAPY, 2024, 32 (04) : 147 - 147
[23] Gene Therapy for Neurological Disease: State of the Art and Opportunities for Next-generation Approaches
Morris, Gareth
Schorge, Stephanie
NEUROSCIENCE, 2022, 490 : 309 - 314
[24] Next-Generation Gene Therapy for Parkinson's Disease Using Engineered Viral Vectors
Bjorklund, Tomas
Davidsson, Marcus
JOURNAL OF PARKINSONS DISEASE, 2021, 11 : S209 - S217
[25] Disease correction by combined neonatal intracranial AAV and systemic lentiviral gene therapy in Sanfilippo Syndrome type B mice
Heldermon, C. D.
Qin, E. Y.
Ohlemiller, K. K.
Herzog, E. D.
Brown, J. R.
Vogler, C.
Hou, W.
Orrock, J. L.
Crawford, B. E.
Sands, M. S.
GENE THERAPY, 2013, 20 (09) : 913 - 921
[26] Disease correction by combined neonatal intracranial AAV and systemic lentiviral gene therapy in Sanfilippo Syndrome type B mice
C D Heldermon
E Y Qin
K K Ohlemiller
E D Herzog
J R Brown
C Vogler
W Hou
J L Orrock
B E Crawford
M S Sands
Gene Therapy, 2013, 20 : 913 - 921
[27] Intravenous AAV9-mediated gene therapy ameliorates neuronal disease of mucopolysaccharidosis type II (MPSII)
Miwa, S.
Shimada, Y.
Higuchi, T.
Kobayashi, H.
Ohashi, T.
HUMAN GENE THERAPY, 2018, 29 (12) : A125 - A126
[28] Gene therapy with AAV-S1S3 improves disease in mucolipidosis type II mice
Dickson, Patricia I.
Srnak, Jennifer
Le, Steven Q.
Sorensen, Alexander
Reardon, Kate
Doray, Balraj
Maloney, Susan
Liu, Lin
Kornfeld, Stuart
MOLECULAR GENETICS AND METABOLISM, 2024, 141 (02)
[29] AAV gene therapy LYS-SAF302 demonstrates widespread sulfamidase distribution in primate brain and correction of disease pathology in MPS IIIA mice
Laufer, Ralph
Hocquemiller, Michael
Hemsley, Kim
MOLECULAR GENETICS AND METABOLISM, 2019, 126 (02) : S91 - S92
[30] Isolation of Next-Generation Gene Therapy Vectors through Engineering, Barcoding, and Screening of Adeno-Associated Virus (AAV) Capsid Variants
Rapti, Kleopatra
Maiakovska, Olena
Becker, Jonas
Szumska, Joanna
Zayas, Margarita
Bubeck, Felix
Liu, Jixin
Gerstmann, Emma
Kraemer, Chiara
Wiedtke, Ellen
Grimm, Dirk
JOVE-JOURNAL OF VISUALIZED EXPERIMENTS, 2022, (188):

← 1 2 3 4 5 →