Prevention of spontaneous bleeding in dogs with haemophilia A and haemophilia B

被引:12
|
作者
Nichols, T. C. [1 ]
Raymer, R. A.
Franck, H. W. G.
Merricks, E. P.
Bellinger, D. A.
Defriess, N.
Margaritis, P. [2 ,3 ]
Arruda, V. R. [2 ,3 ]
Kay, M. A. [4 ]
High, K. A. [2 ,3 ,5 ]
机构
[1] Univ N Carolina, UNC Sch Med, Dept Pathol & Lab Med, Francis Owen Blood Res Lab, Chapel Hill, NC 27516 USA
[2] Univ Penn, Dept Pediat, Med Ctr, Philadelphia, PA 19104 USA
[3] Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA
[4] Stanford Univ, Dept Pediat, Program Human Gene Therapy, Stanford, CA 94305 USA
[5] Howard Hughes Med Inst, Dept Pediat, Philadelphia, PA USA
关键词
dogs; haemophilia A; haemophilia B; spontaneous bleeding; FACTOR-VIII GENE; ADENOASSOCIATED VIRAL VECTOR; ANTIHEMOPHILIC-FACTOR AHF; LONG-TERM CORRECTION; HUMAN-FACTOR-IX; CANINE HEMOPHILIA; VONWILLEBRAND DISEASE; REPLACEMENT THERAPY; SKELETAL-MUSCLE; MUTATION;
D O I
10.1111/j.1365-2516.2010.02255.x
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
Dogs with haemophilia A or haemophilia B exhibit spontaneous bleeding comparable with the spontaneous bleeding phenotype that occurs in humans with severe haemophilia. The phenotypic and genotypic characteristics of haemophilic dogs have been well-described, and such dogs are suitable for testing prophylactic protein replacement therapy and gene transfer strategies. In dogs with haemophilia, long-term effects on spontaneous bleeding frequency (measured over years) can be used as an efficacy endpoint in such studies. Although complete correction of coagulopathy has not been achieved, published data show that prophylactic factor replacement therapy and gene transfer can markedly reduce the frequency of spontaneous bleeding in haemophilic dogs. Further studies are currently ongoing.
引用
收藏
页码:19 / 23
页数:5
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