A new "FIX" for hemophilia B gene therapy

被引：3

作者：

Almeida-Porada, Graca ^{[1
]}

机构：

[1] Wake Forest Sch Med, Winston Salem, NC 27101 USA

来源：

BLOOD | 2021年 / 137卷 / 21期

关键词：

FACTOR-IX;

D O I：

10.1182/blood.2021011753

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

In this issue of Blood, Nair et al(1) demonstrate that AAV-directed gene therapy using a new bioengineered FIX transgene provides higher FIX activity and superior hemostatic efficacy than other FIX variants and may allow for lower and potentially safer vector doses in future human clinical trials.

引用

页码：2860 / 2861

页数：3

共 7 条

[1] Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant [J].

George, L. A. ;

Sullivan, S. K. ;

Giermasz, A. ;

Rasko, J. E. J. ;

Samelson-Jones, B. J. ;

Ducore, J. ;

Cuker, A. ;

Sullivan, L. M. ;

Majumdar, S. ;

Teitel, J. ;

McGuinn, C. E. ;

Ragni, M. V. ;

Luk, A. Y. ;

Hui, D. ;

Wright, J. F. ;

Chen, Y. ;

Liu, Y. ;

Wachtel, K. ;

Winters, A. ;

Tiefenbacher, S. ;

Arruda, V. R. ;

van der Loo, J. C. M. ;

Zelenaia, O. ;

Takefman, D. ;

Carr, M. E. ;

Couto, L. B. ;

Anguela, X. M. ;

High, K. A. .

NEW ENGLAND JOURNAL OF MEDICINE, 2017, 377 (23) :2215-2227

[2] AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B [J].

Manno, CS ;

Chew, AJ ;

Hutchison, S ;

Larson, PJ ;

Herzog, RW ;

Arruda, VP ;

Tai, SJ ;

Ragni, MV ;

Thompson, A ;

Ozelo, M ;

Couto, LB ;

Leonard, DGB ;

Johnson, FA ;

McClelland, A ;

Scallan, C ;

Skarsgard, E ;

Flake, AW ;

Kay, MA ;

High, KA ;

Glader, B .

BLOOD, 2003, 101 (08) :2963-2972

[3] Successful transduction of liver in hemophilia by AAV-factor IX and limitations imposed by the host immune response [J].

Manno, CS ;

Arruda, VR ;

Pierce, GF ;

Glader, B ;

Ragni, M ;

Rasko, J ;

Ozelo, MC ;

Hoots, K ;

Blatt, P ;

Konkle, B ;

Dake, M ;

Kaye, R ;

Razavi, M ;

Zajko, A ;

Zehnder, J ;

Nakai, H ;

Chew, A ;

Leonard, D ;

Wright, JF ;

Lessard, RR ;

Sommer, JM ;

Tigges, M ;

Sabatino, D ;

Luk, A ;

Jiang, HY ;

Mingozzi, F ;

Couto, L ;

Ertl, HC ;

High, KA ;

Kay, MA .

NATURE MEDICINE, 2006, 12 (03) :342-347

[4] Immune responses to AAV in clinical trials [J].

Mingozzi, Federico ;

High, Katherine A. .

CURRENT GENE THERAPY, 2007, 7 (05) :316-324

[5] Gene therapy for hemophilia B using CB 2679d-GT: a novel factor IX variant with higher potency than factor IX Padua [J].

Nair, Nisha ;

De Wolf, Dries ;

Phuong Anh Nguyen ;

Quang Hong Pham ;

Samara-Kuko, Ermira ;

Landau, Jeff ;

Blouse, Grant E. ;

Chuah, Marinee K. ;

VandenDriessche, Thierry .

BLOOD, 2021, 137 (21) :2902-2906

[6] Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B [J].

Nathwani, A. C. ;

Reiss, U. M. ;

Tuddenham, E. G. D. ;

Rosales, C. ;

Chowdary, P. ;

McIntosh, J. ;

Della Peruta, M. ;

Lheriteau, E. ;

Patel, N. ;

Raj, D. ;

Riddell, A. ;

Pie, J. ;

Rangarajan, S. ;

Bevan, D. ;

Recht, M. ;

Shen, Y. -M. ;

Halka, K. G. ;

Basner-Tschakarjan, E. ;

Mingozzi, F. ;

High, K. A. ;

Allay, J. ;

Kay, M. A. ;

Ng, C. Y. C. ;

Zhou, J. ;

Cancio, M. ;

Morton, C. L. ;

Gray, J. T. ;

Srivastava, D. ;

Nienhuis, A. W. ;

Davidoff, A. M. .

NEW ENGLAND JOURNAL OF MEDICINE, 2014, 371 (21) :1994-2004

[7] X-Linked Thrombophilia with a Mutant Factor IX (Factor IX Padua) [J].

Simioni, Paolo ;

Tormene, Daniela ;

Tognin, Giulio ;

Gavasso, Sabrina ;

Bulato, Cristiana ;

Iacobelli, Nicholas P. ;

Finn, Jonathan D. ;

Spiezia, Luca ;

Radu, Claudia ;

Arruda, Valder R. .

NEW ENGLAND JOURNAL OF MEDICINE, 2009, 361 (17) :1671-1675

← 1 →