JPWO2023106261A5 - - Google Patents

Download PDF

Info

Publication number
JPWO2023106261A5
JPWO2023106261A5 JP2023566309A JP2023566309A JPWO2023106261A5 JP WO2023106261 A5 JPWO2023106261 A5 JP WO2023106261A5 JP 2023566309 A JP2023566309 A JP 2023566309A JP 2023566309 A JP2023566309 A JP 2023566309A JP WO2023106261 A5 JPWO2023106261 A5 JP WO2023106261A5
Authority
JP
Japan
Prior art keywords
amino acid
acid sequence
promoter
associated virus
seq
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2023566309A
Other languages
English (en)
Japanese (ja)
Other versions
JPWO2023106261A1 (https=
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/JP2022/044719 external-priority patent/WO2023106261A1/ja
Publication of JPWO2023106261A1 publication Critical patent/JPWO2023106261A1/ja
Publication of JPWO2023106261A5 publication Critical patent/JPWO2023106261A5/ja
Pending legal-status Critical Current

Links

JP2023566309A 2021-12-06 2022-12-05 Pending JPWO2023106261A1 (https=)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
JP2021197789 2021-12-06
PCT/JP2022/044719 WO2023106261A1 (ja) 2021-12-06 2022-12-05 鉄蓄積性神経変性疾患の治療のための組換えアデノ随伴ウイルスベクター

Publications (2)

Publication Number Publication Date
JPWO2023106261A1 JPWO2023106261A1 (https=) 2023-06-15
JPWO2023106261A5 true JPWO2023106261A5 (https=) 2024-10-10

Family

ID=86730357

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023566309A Pending JPWO2023106261A1 (https=) 2021-12-06 2022-12-05

Country Status (4)

Country Link
US (1) US20250312484A1 (https=)
EP (1) EP4446419A1 (https=)
JP (1) JPWO2023106261A1 (https=)
WO (1) WO2023106261A1 (https=)

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6506559B1 (en) 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
ES2336887T5 (es) 2000-03-30 2019-03-06 Whitehead Inst Biomedical Res Mediadores de interferencia por ARN específicos de secuencias de ARN
WO2003093479A1 (en) 2002-05-01 2003-11-13 University Of Florida Research Foundation, Inc. Improved raav expression systems and methods for enhancing transduction of mammalian neural cells
ES2714007T3 (es) 2007-04-09 2019-05-24 Univ Florida Composiciones de vectores rAAV que tienen proteínas de la cápside modificadas en tirosina y métodos para su uso
WO2012057363A1 (ja) 2010-10-27 2012-05-03 学校法人自治医科大学 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン
WO2013174760A1 (en) 2012-05-25 2013-11-28 Deutsches Krebsforschungszentrum Optimized aav-vectors for high transduction rates in dendritic cells

Similar Documents

Publication Publication Date Title
Lundberg et al. Applications of lentiviral vectors for biology and gene therapy of neurological disorders
JP5907579B2 (ja) 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン
TWI835761B (zh) 工程化dna結合蛋白
CN117321213A (zh) 具有优选表达水平的腺相关病毒组合物
CN102459611B (zh) 神经退行性疾病的基因治疗
CN107532173A (zh) 靶向中枢神经系统的aav载体
KR20240110849A (ko) Aadc/gdnf 폴리뉴클레오티드 및 이의 파킨슨병 치료에 있어서의 용도
Carlsson et al. Restoration of the striatal dopamine synthesis for Parkinson's disease: viral vector-mediated enzyme replacement strategy
CN113227126A (zh) 成纤维细胞生长因子21(fgf21)基因治疗
JP7334996B2 (ja) グルコーストランスポーター１発現用アデノ随伴ウイルスベクター
JPWO2023106261A5 (https=)
JP7641911B2 (ja) 新型の酵素組成物
CN117106825B (zh) 用于治疗帕金森病的基因治疗载体及其用途
CA3197316A1 (en) Neurod1 and dlx2 vector
WO2021031025A1 (zh) Ptbp1抑制剂在预防和/或治疗神经退行性疾病中的应用
ES2883358T3 (es) Gen Dgkk recombinante para terapia génica del síndrome de X frágil
US20220098616A1 (en) ISL1 and LHX3 VECTOR
US20220098617A1 (en) Ascl1 vector
US20260002176A1 (en) Adeno-associated virus gene therapy products and methods
HK40095433A (zh) 用於治疗帕金森病的基因治疗载体及其用途
HK40095433B (zh) 用於治疗帕金森病的基因治疗载体及其用途
CN121866337A (zh) 经修饰的腺相关病毒载体及其在治疗神经疾病中的用途
TW202415773A (zh) 用於治療神經系統疾病的ａａｖ基因療法
WO2025090858A1 (en) Methods for identifying aav capsid variants with desired characteristics
CN116710566A (zh) Neurod1载体