Gene Therapy Approaches for the Treatment of Hemophilia B

被引：16

作者：

Soroka, Anastasiia B. B. ^{[1
]}

Feoktistova, Sofya G. G. ^{[1
]}

Mityaeva, Olga N. N. ^{[1
]}

Volchkov, Pavel Y. Y. ^{[1
]}

机构：

[1] Natl Res Univ, Moscow Inst Phys & Technol, Life Sci Res Ctr, Dolgoprudnyi 141700, Russia

来源：

INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES | 2023年 / 24卷 / 13期

基金：

俄罗斯科学基金会;

关键词：

hemophilia B; gene therapy; AAV; genome editing; blood coagulation factor IX; hemostasis; LONG-TERM SAFETY; IN-VIVO; ALBUMIN LOCUS; VIRUS VECTOR; AAV; EFFICACY; FIX; ANTITHROMBIN; CRISPR/CAS9; HEMOSTASIS;

D O I：

10.3390/ijms241310766

中图分类号：

Q5 [生物化学]; Q7 [分子生物学];

学科分类号：

071010 ; 081704 ;

摘要：

In contrast to the standard enzyme-replacement therapy, administered from once per 7-14 days to 2-3 times a week in patients with severe hemophilia B, as a result of a single injection, gene therapy can restore F9 gene expression and maintain it for a prolonged time. In clinical research, the approach of delivering a functional copy of a gene using adeno-associated viral (AAV) vectors is widely used. The scientific community is actively researching possible modifications to improve delivery efficiency and expression. In preclinical studies, the possibility of genome editing using CRISPR/Cas9 technology for the treatment of hemophilia B is also being actively studied.

引用

页数：17

共 77 条

[1] A new "FIX" for hemophilia B gene therapy [J].

Almeida-Porada, Graca .

BLOOD, 2021, 137 (21) :2860-2861

[2] Search-and-replace genome editing without double-strand breaks or donor DNA [J].

Anzalone, Andrew V. ;

Randolph, Peyton B. ;

Davis, Jessie R. ;

Sousa, Alexander A. ;

Koblan, Luke W. ;

Levy, Jonathan M. ;

Chen, Peter J. ;

Wilson, Christopher ;

Newby, Gregory A. ;

Raguram, Aditya ;

Liu, David R. .

NATURE, 2019, 576 (7785) :149-+

[3] Gene therapy for immune tolerance induction in hemophilia with inhibitors [J].

Arruda, V. R. ;

Samelson-Jones, B. J. .

JOURNAL OF THROMBOSIS AND HAEMOSTASIS, 2016, 14 (06) :1121-1134

[4] Designer nuclease-mediated gene correction via homology-directed repair in an in vitro model of canine hemophilia B [J].

Bergmann, Thorsten ;

Ehrke-Schulz, Eric ;

Gao, Jian ;

Schiwon, Maren ;

Schildgen, Verena ;

David, Stephan ;

Schildgen, Oliver ;

Ehrhardt, Anja .

JOURNAL OF GENE MEDICINE, 2018, 20 (05)

[5] Clinical, humanistic, and economic burden of severe haemophilia B in adults receiving factor IX prophylaxis: findings from the CHESS II real-world burden of illness study in Europe [J].

Burke, Tom ;

Asghar, Sohaib ;

O'Hara, Jamie ;

Chuang, Margaret ;

Sawyer, Eileen K. ;

Li, Nanxin .

ORPHANET JOURNAL OF RARE DISEASES, 2021, 16 (01)

[6] Clinical, humanistic, and economic burden of severe hemophilia B in the United States: Results from the CHESS US and CHESS US plus population surveys [J].

Burke, Tom ;

Asghar, Sohaib ;

O'Hara, Jamie ;

Sawyer, Eileen K. ;

Li, Nanxin .

ORPHANET JOURNAL OF RARE DISEASES, 2021, 16 (01)

[7] Long-Term Safety and Efficacy of Nonacog Beta Pegol (N9-GP) Administered for at Least 5 Years in Previously Treated Children with Hemophilia B [J].

Carcao, Manuel ;

Kearney, Susan ;

Lu, Meng Yao ;

Taki, Masashi ;

Rubens, Daniel ;

Shen, Chunduo ;

Santagostino, Elena .

THROMBOSIS AND HAEMOSTASIS, 2020, 120 (05) :737-746

[8] Enhanced proofreading governs CRISPR-Cas9 targeting accuracy [J].

Chen, Janice S. ;

Dagdas, Yavuz S. ;

Kleinstiver, Benjamin P. ;

Welch, Moira M. ;

Sousa, Alexander A. ;

Harrington, Lucas B. . ;

Sternberg, Samuel H. ;

Joung, J. Keith ;

Yildiz, Ahmet ;

Doudna, Jennifer A. .

NATURE, 2017, 550 (7676) :407-+

[9] CRISPR/Cas9-mediated knockin of human factor IX into swine factor IX locus effectively alleviates bleeding in hemophilia B pigs [J].

Chen, Jiahuan ;

An, Beiying ;

Yu, Biao ;

Peng, Xiaohuan ;

Yuan, Hongming ;

Yang, Qiangbing ;

Chen, Xue ;

Yu, Tingting ;

Wang, Lingyu ;

Zhang, Xinwei ;

Wang, He ;

Zou, Xiaodong ;

Pang, Daxin ;

Ouyang, Hongsheng ;

Tang, Xiaochun .

HAEMATOLOGICA, 2021, 106 (03) :829-837

[10] Phase 1-2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B [J].

Chowdary, Pratima ;

Shapiro, Susan ;

Makris, Mike ;

Evans, Gillian ;

Boyce, Sara ;

Talks, Kate ;

Dolan, Gerard ;

Reiss, Ulrike ;

Phillips, Mark ;

Riddell, Anne ;

Peralta, Maria R. ;

Quaye, Michelle ;

Patch, David W. ;

Tuddenham, Edward ;

Dane, Allison ;

Watissee, Marie ;

Long, Alison ;

Nathwani, Amit .

NEW ENGLAND JOURNAL OF MEDICINE, 2022, 387 (03) :237-247

← 1 2 3 4 5 6 7 8 →