Human clotting factor IX (hF IX) secretion in goat milk after direct transfer with hF IX minigene into mammary gland by using retroviral vectors

1997 ◽  
Vol 42 (15) ◽  
pp. 1308-1313 ◽  
Author(s):  
Kezhong Zhang ◽  
Hongwei Wang ◽  
Yun Bao ◽  
Daru Lu ◽  
Jinglun Xue ◽  
...  
Keyword(s):  
Mammary Gland ◽  
Goat Milk ◽  
Retroviral Vectors ◽  
Factor Ix ◽  
Clotting Factor ◽  
Direct Transfer ◽  
Human Clotting Factor

Expression of biologically active human clotting factor IX (hF IX) in the mammary gland of transgenic mice

1998 ◽  
Vol 43 (15) ◽  
pp. 1294-1298 ◽  
Author(s):  
Ying Huang ◽  
Kezhong Zhang ◽  
Wenying Huang ◽  
Daru Lu ◽  
Ying Huang ◽  
...  
Keyword(s):  
Mammary Gland ◽  
Transgenic Mice ◽  
Factor Ix ◽  
Biologically Active ◽  
Clotting Factor ◽  
Human Clotting Factor

scholarly journals Production of human factor IX in animals by genetically modified skin fibroblasts: potential therapy for hemophilia B

Blood ◽  
1989 ◽  
Vol 73 (2) ◽  
pp. 438-445
Author(s):  
TD Palmer ◽  
AR Thompson ◽  
AD Miller
Keyword(s):  
Human Factor ◽  
Normal Skin ◽  
Human Fibroblasts ◽  
Retroviral Vectors ◽  
Factor Ix ◽  
Skin Fibroblasts ◽  
Hemophilia B ◽  
Clotting Factor ◽  
Human Factor Ix

Inherited diseases might be treated by introducing normal genes into a patient's somatic tissues to correct the genetic defects. In the case of hemophilia resulting from a missing clotting factor, the required gene could be introduced into any cell as long as active factor reached the circulation. We previously showed that retroviral vectors can efficiently transfer genes into normal skin fibroblasts and that the infected cells can produce high levels of a therapeutic product in vitro. In the current study, we examined the ability of skin fibroblasts to secrete active clotting factor after infection with different retroviral vectors encoding human clotting factor IX. Normal human fibroblasts infected with one vector secreted greater than 3 micrograms factor IX/10(6) cells/24 h. Of this protein, greater than 70% was structurally and functionally indistinguishable from human factor IX derived from normal plasma. This suggests that infected autologous fibroblasts might provide therapeutic levels of factor IX if transplanted into patients suffering from hemophilia B. By transplanting normal diploid fibroblasts infected with the factor IX vectors, we showed that human factor IX can be produced and is circulated at readily detectable levels in rats and mice.

scholarly journals Production of human factor IX in animals by genetically modified skin fibroblasts: potential therapy for hemophilia B

Blood ◽  
1989 ◽  
Vol 73 (2) ◽  
pp. 438-445 ◽  
Author(s):  
TD Palmer ◽  
AR Thompson ◽  
AD Miller
Keyword(s):  
Human Factor ◽  
Normal Skin ◽  
Human Fibroblasts ◽  
Retroviral Vectors ◽  
Factor Ix ◽  
Skin Fibroblasts ◽  
Hemophilia B ◽  
Clotting Factor ◽  
Human Factor Ix

Abstract Inherited diseases might be treated by introducing normal genes into a patient's somatic tissues to correct the genetic defects. In the case of hemophilia resulting from a missing clotting factor, the required gene could be introduced into any cell as long as active factor reached the circulation. We previously showed that retroviral vectors can efficiently transfer genes into normal skin fibroblasts and that the infected cells can produce high levels of a therapeutic product in vitro. In the current study, we examined the ability of skin fibroblasts to secrete active clotting factor after infection with different retroviral vectors encoding human clotting factor IX. Normal human fibroblasts infected with one vector secreted greater than 3 micrograms factor IX/10(6) cells/24 h. Of this protein, greater than 70% was structurally and functionally indistinguishable from human factor IX derived from normal plasma. This suggests that infected autologous fibroblasts might provide therapeutic levels of factor IX if transplanted into patients suffering from hemophilia B. By transplanting normal diploid fibroblasts infected with the factor IX vectors, we showed that human factor IX can be produced and is circulated at readily detectable levels in rats and mice.

Efficient transfer and expression of human clotting factor IX cDNA in neonatal hemophilia B mice mediated by VSV-G pseudotyped retrovirus

2001 ◽  
Vol 46 (18) ◽  
pp. 1534-1538
Author(s):  
Hongwei Wang ◽  
Chenbo Ye ◽  
Li Chen ◽  
Xuefeng Wang ◽  
Xinfang Qiu ◽  
...  
Keyword(s):  
Factor Ix ◽  
Hemophilia B ◽  
Clotting Factor ◽  
B Mice ◽  
Human Clotting Factor ◽  
Efficient Transfer

Sustained delivery of therapeutic concentrations of human clotting factor IX - a comparison of adenoviral and AAV vectors administeredin utero

2002 ◽  
Vol 4 (1) ◽  
pp. 46-53 ◽  
Author(s):  
Holm Schneider ◽  
Christiane Mühle ◽  
Anne Marie Douar ◽  
Simon Waddington ◽  
Qiu-Jie Jiang ◽  
...  
Keyword(s):  
Factor Ix ◽  
Clotting Factor ◽  
Sustained Delivery ◽  
Human Clotting Factor

Long-Term Expression of Human Clotting Factor IX from Retrovirally Transduced Primary Human KeratinocytesIn Vivo

1998 ◽  
Vol 9 (8) ◽  
pp. 1187-1195 ◽  
Author(s):  
Steve J. White ◽  
Sean M. Page ◽  
Paris Margaritis ◽  
George G. Brownlee
Keyword(s):  
Factor Ix ◽  
Clotting Factor ◽  
Human Clotting Factor
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