Hemophilia A: an ideal disease to correct in utero.
Hemophilia A: an ideal disease to correct in utero.
复制标题
血友病A：一种理想的子宫内疾病。
DOI：
10.3389/fphar.2014.00276
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发表时间：
2014
影响因子：
5.6
通讯作者：
Almeida-Porada G
中科院分区：
文献类型：
--
作者：
Porada CD;Rodman C;Ignacio G;Atala A;Almeida-Porada G
Hemophilia A (HA) is the most frequent inheritable defect of the coagulation proteins. The current standard of care for patients with HA is prophylactic factor infusion, which is comprised of regular (2–3 times per week) intravenous infusions of recombinant or plasma-derived FVIII to maintain hemostasis. While this treatment has greatly increased the quality of life and lengthened the life expectancy for many HA patients, its high cost, the need for lifelong infusions, and the fact that it is unavailable to roughly 75% of the world's HA patients make this type of treatment far from ideal. In addition, this lifesaving therapy suffers from a high risk of treatment failure due to immune response to the infused FVIII. There is thus a need for novel treatments, such as those using stem cells and/or gene therapy, which have the potential to mediate long-term correction or permanent cure following a single intervention. In the present review, we discuss the clinical feasibility and unique advantages that an in utero approach to treating HA could offer, placing special emphasis on a new sheep model of HA we have developed and on the use of mesenchymal stromal cells (MSC) as cellular vehicles for delivering the FVIII gene.
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影响因子：
4.2
作者：
Bartholomew, A;Patil, S;Devine, SM
通讯作者：
Devine, SM
DOI：
10.1098/rspb.1954.0054
发表时间：
1954-01-01
期刊：
PROCEEDINGS OF THE ROYAL SOCIETY SERIES B-BIOLOGICAL SCIENCES
影响因子：
--
作者：
BILLINGHAM, RE;BRENT, L;MEDAWAR, PB
通讯作者：
MEDAWAR, PB
影响因子：
2.1
作者：
Almeida-Porada, G;Porada, C;Zanjani, ED
通讯作者：
Zanjani, ED
影响因子：
5.2
作者：
Banas, Agnieszka;Teratani, Takumi;Ochiya, Takahiro
通讯作者：
Ochiya, Takahiro
影响因子：
24.5
作者：
Aurich, Ines;Mueller, Lutz P.;Christ, Bruno
通讯作者：
Christ, Bruno