You can get a Fast Pass at Disneyland with a Hemophilia Hospital card and it will allow you and your child to have access to almost all the rides line-free.
Just stop by Disney City Hall.
Just stop by Disney City Hall.
The whole thing about having a surgery for my little one worries and scares me so much! I have been doing my best to prolong NOT having the surgery to put a port... what if tomorrow there will be THE cure or new factor that lasts longer will become available?
However, after the last few days of my son being poked 7 (SEVEN) times, the realization that we will have to have surgery sooner than later is evident. What my little Prince had to deal with in the last few days is not acceptable!
However, after the last few days of my son being poked 7 (SEVEN) times, the realization that we will have to have surgery sooner than later is evident. What my little Prince had to deal with in the last few days is not acceptable!
Along my life path I've learned that learning from others and sharing is the most powerful thing. If one fails...learn how and avoid those mistakes if possible. If one succeeds... learn how and do what you can to replicate it in your life.
To learn from others and to receive up-to-date information, I've signed up for several enewsletters and "to receive information". Today, I got a a package from Red Chip Enterprises with many different books for me (as a parent) and for my son to help us live a better more informative life.
Here are the titles of the books that just arrived:
To learn from others and to receive up-to-date information, I've signed up for several enewsletters and "to receive information". Today, I got a a package from Red Chip Enterprises with many different books for me (as a parent) and for my son to help us live a better more informative life.
Here are the titles of the books that just arrived:
- What to expect during the Primary School years
- Teaching the Educators
- Just a Boy
- Dental Care
- Alexis, The Prince Who Had Hemophilia
- How Children Understand Hemophilia
- A practical guide
- Teach Your Child
Josh walk up today very clingy. I thought he just missed his mommy since I was not able to spent time with him yesterday as I was sick and didn't want him to catch it. But something inside of me felt unease. Not that I mind extra hugs, quite contrary, I would spent the whole day cuddle up with my baby, but something seemed wrong.
When I started to put his t-shirt on, it became evident. He favor his left arm and he was showing me that his right arm was hurting him. There was no bruise, no swelling, nothing that would alarm me. I've learned, when in doubt, call! So I called Children's Hospital immediately. We are on our way. Poor Baby is going to get pocked again. :-(
When I started to put his t-shirt on, it became evident. He favor his left arm and he was showing me that his right arm was hurting him. There was no bruise, no swelling, nothing that would alarm me. I've learned, when in doubt, call! So I called Children's Hospital immediately. We are on our way. Poor Baby is going to get pocked again. :-(
An Open Letter To A New Hemom... Tatyana Hi Tatyana,
No need to thank us. We thank you for finding us and we are so grateful to have made a difference in your life.
I guess, I'll start by saying, we all had the same fears when we first came about the news that our precious children are affected by the condition... Hemophilia.
For years, I've tried to find answers too. Tried so hard to get understanding for it. It took me a while, nevertheless, worth every effort. Through the help of my son's medical team, treatment centers, other Hemoms, volunteers and the entire hemophilia community, our family was able to survive the nightmare. I always thank God for that! I always thank Him for guiding us in our way through our darkest moments.
My son now is in college. He was diagnosed with severe Hemophilia A at age seven. Currently a 3x National champion in golf, living a normal life through prophylaxis treatment of his meds, Helixate FS. Never better... We are so proud of him!!!
We Hemoms are here for you. We are here for each other. Feel free to ask questions and each one will be able to help guide you through your journey, as they have for mine. We have a website that you can always visit as well with our stories, at www.hemophiliamoms.com. I'm sure you'll find the site not just informative, but also full of resources. I guarantee you that it will give you, not only peace of mind and assurance, but also the greatest gift you can possibly imagine... Hope.
Take care,
Jenny Posted by Jenny at 10:20 PM 1 comments: Suzanne said... Tatyana,
I hope you find comfort in knowing you are not on this course alone. Jenny gave you the best advice there is....get connected. My 10 year old son was a spontaneous mutuation and was diagnosed at birth. Finding and getting involved in our local Treatment Center, local Hemophilia Chapter, support groups and this web site saved me. I just said the other day that "It takes a village to raise a child" and no truer words have been spoken. I am blessed with a very supportive family but it just isn't enough sometimes! I needed support, encouragement and advice from those who have walked a mile in my shoes. Understand the fears and anxiety we are experiencing as Mom's and can encourage through experience. Thank you for sharing your story with us and we would love to hear from you. My son leads a normal life thanks to modern medicines like Helixate FS. He plays baseball, golf, basketball, swims and is active in almost every area in school. He's a boy. Welcome to our family of Hemophilia Moms.
www.hemophiliamoms.blogspot.com
No need to thank us. We thank you for finding us and we are so grateful to have made a difference in your life.
I guess, I'll start by saying, we all had the same fears when we first came about the news that our precious children are affected by the condition... Hemophilia.
For years, I've tried to find answers too. Tried so hard to get understanding for it. It took me a while, nevertheless, worth every effort. Through the help of my son's medical team, treatment centers, other Hemoms, volunteers and the entire hemophilia community, our family was able to survive the nightmare. I always thank God for that! I always thank Him for guiding us in our way through our darkest moments.
My son now is in college. He was diagnosed with severe Hemophilia A at age seven. Currently a 3x National champion in golf, living a normal life through prophylaxis treatment of his meds, Helixate FS. Never better... We are so proud of him!!!
We Hemoms are here for you. We are here for each other. Feel free to ask questions and each one will be able to help guide you through your journey, as they have for mine. We have a website that you can always visit as well with our stories, at www.hemophiliamoms.com. I'm sure you'll find the site not just informative, but also full of resources. I guarantee you that it will give you, not only peace of mind and assurance, but also the greatest gift you can possibly imagine... Hope.
Take care,
Jenny Posted by Jenny at 10:20 PM 1 comments: Suzanne said... Tatyana,
I hope you find comfort in knowing you are not on this course alone. Jenny gave you the best advice there is....get connected. My 10 year old son was a spontaneous mutuation and was diagnosed at birth. Finding and getting involved in our local Treatment Center, local Hemophilia Chapter, support groups and this web site saved me. I just said the other day that "It takes a village to raise a child" and no truer words have been spoken. I am blessed with a very supportive family but it just isn't enough sometimes! I needed support, encouragement and advice from those who have walked a mile in my shoes. Understand the fears and anxiety we are experiencing as Mom's and can encourage through experience. Thank you for sharing your story with us and we would love to hear from you. My son leads a normal life thanks to modern medicines like Helixate FS. He plays baseball, golf, basketball, swims and is active in almost every area in school. He's a boy. Welcome to our family of Hemophilia Moms.
www.hemophiliamoms.blogspot.com
Hemophilia is a bleeding disorder caused by the absence of an essential blood clotting protein. He will always have the same level of factor deficiency he is born with. Check out Myths about Hemophilia.
Vitamin K-Rich Foods Foods rich in vitamin K can help platelets function properly. Vitamin K plays an essential role in platelet function and blood coagulation, helping to regulate the enzymes required for blood clot formation. The Linus Pauling Institute at Oregon State University explains that vitamin K helps activate seven proteins involved in the clotting cascade. Foods rich in vitamin K include leafy greens like lettuce and parsley, olive, soybean and canola oils as well as cruciferous vegetables like broccoli, kale and Swiss chard.
Read more: http://www.livestrong.com/article/357821-what-foods-help-platelets/#ixzz1G57qGcwL
Read more: http://www.livestrong.com/article/357821-what-foods-help-platelets/#ixzz1G57qGcwL
Josh had his first nose bleed yesterday. It is always scary when you see your child's face covered with blood, it's even worse when a child has Hemophilia. Of course my first reaction is AHHHHHHHH!!!! I AM FREAKING OUT! And those High School Drama classes really pay off when you freak out inside and have to show a calm and knowledgeable face to your baby.
My husband ran to call the doctor to check what to do.
Meanwhile, with my years of "mommy-doctor" training, I thought that bath can sooth his nose bleed. Josh was thrilled with the idea of going to the bath, after all it's his favorite activity. Who wouldn't love getting everything and their mommy completely soaking wet?
The bleeding seemed to subside.
My husband came to the room to share what the doctor told him. If the bleeding will stop in 3 minutes or less, then it's common "vein-pop" bleed and we don't have to run to the hospital.
Saline helps as well to sooth the nose bleed.
Here is an article I found:
How to stop a nosebleed in someone with hemophilia A:
My husband ran to call the doctor to check what to do.
Meanwhile, with my years of "mommy-doctor" training, I thought that bath can sooth his nose bleed. Josh was thrilled with the idea of going to the bath, after all it's his favorite activity. Who wouldn't love getting everything and their mommy completely soaking wet?
The bleeding seemed to subside.
My husband came to the room to share what the doctor told him. If the bleeding will stop in 3 minutes or less, then it's common "vein-pop" bleed and we don't have to run to the hospital.
Saline helps as well to sooth the nose bleed.
Here is an article I found:
How to stop a nosebleed in someone with hemophilia A:
- Pinch the nose firmly for 15 minutes without letting go. Make sure you are pinching the bulb of the nose so that it is completely closed.
- Spray decongestant nasal spray in each nostril prior to the nose pinch. Do not use a decongestant if you have heart disease or high blood pressure.
- Sit upright, do not lie down.
- Breathe through your mouth.
- Drink cold water to clear blood from your throat.
- Seek medical care if the bleeding does not stop within 15 to 20 minutes.
- Stop smoking.
- Avoid hot liquids.
- Do not take aspirin for at least one week.
- Do not pick blood from your nose once the bleeding has stopped.
- Do not blow your nose for 2 days
Thousands of years of suffering, with no effective treatment for Hemophilia, and then in the last few decades research has exploded.
Hemophilia Research1965 Cryoprecipitate used to treat Hemophilia.1
1968 First FIX and FVIII Concentrates.1
1982 FIX Gene cloned.1
1982 FVIII Gene cloned.1
1985 First viral inactivated factor concentrates become available.1
1992 First Recombinant product becomes available.1
1992 First Gene Therapy trials in mice.1
1993-1994 FIX First Gene Therapy in dogs.1
1993 FIX First Gene Therapy in humans 2% factor level increase.
1994 FIX Gene Therapy in dogs up to 300% increase for 1-2 months.2
1995-1996 First FIX Gene therapy in mice and dogs.1
1996 FVIII Gene Therapy in dogs well over therapeutic level for 1-2 weeks.31997 First Recombinant FIX product.1
1998 FVIII Gene Therapy in humans 4% factor level increase.1
1999 FIX Gene Therapy in humans B 1.6% increase.1
1999 FVIII Gene Therapy in mice produce 20 % increase for 11 months.4
2000 FVIII Gene Therapy in mice 4%-20% increase.5
2001 FIX Gene Therapy in humans 2%- 12% increase.1
2001 FVIII Gene Therapy in humans up to 3% increase.1
2002 FVIII Gene Therapy in mice up to 20 % increase.6
2003 FIX Gene Therapy in neonatal mice 150%-280% for more than 14 Months.72003 FIX Gene Therapy in neonatal dogs 12%- 36% increase for more than 14 Months.7
2004 FVIII Gene Therapy in mice produces 100% correction.8
2004 Vwb Factor Genetically produced in culture.9
2004 FIX Gene therapy in dogs 4%-14% increase for 3 years, observation ongoing.10
2005 Glannzman's Thrombasthenia Gene Therapy produces therapeutic levels in mice.11
2005 FVIII Gene Therapy in newborn mice and dogs produces 115% - 139% factor levels for 1 1/2 years, observation ongoing.12
2006 Gene Therapy produces FVIII stored in platelets to overcome inhibitors.14
2006 FVIIA Gene Therapy in mice produces therapeutic levels to overcome inhibitors.15
2006 FIX produced from microcapsule implanted in mice.16
2006 FIX Gene Therapy in mice produces Long Term Therapeutic levels.17
2007 FIX Gene Therapy in humans produces increases for over 3.7 years observation ongoing.18
2007 FVIII Gene Therapy in dogs and mice produces factor level increases 2%-4.5% for 27 weeks.19
2007 Vwb Factor produced by Gene Therapy.20
2007 FIX Gene Therapy in mice produces over 10% for more than 280 Days.21
2006 FVIIa Gene Therapy in mice produces FVIIa.13
1 C. Hough, D. Lillicrap (2005) Gene therapy for hemophilia: an imperative to succeed, Journal of Thrombosis and Haemostasis 3 (6), 1195-1205. doi:10.1111/j.1538-7836.2005.01401.x
2 Kay MA, Landen CN, Rothenberg SR, Taylor LA, Leland F, Wiehle S, Fang B, Bellinger D, Finegold M, Thompson AR, et al. In vivo hepatic gene therapy: complete albeit transient correction of factor IX deficiency in hemophilia B dogs.1: Proc Natl Acad Sci U S A. 1994 Mar 15;91(6):2353-7.
3 S Connelly, J Mount, A Mauser, JM Gardner, M Kaleko, A McClelland, and CD Jr Lothrop Complete short-term correction of canine hemophilia A by in vivo gene therapy Blood, Nov 1996; 88: 3846 - 3853.
4 Lang, H Study points to possible cure for hemophilia UNC-CH School of Medicine News Services June 9, 1999--No.369
5 Steven S. Fakharzadeh, Yue Zhang, Rita Sarkar, and Haig H. Kazazian, JrCorrection of the coagulation defect in hemophilia A mice through factor VIII expression in skin Blood, May 2000; 95: 2799 - 2805.
6 Arnette R. New gene therapy treatment works in mice with hemophilia A. UNC School of Medicine News Services June 10, 2002 -- No. 332
7 Lingfei Xu, Cuihua Gao, Mark S. Sands, Shi-Rong Cai, Timothy C. Nichols, Dwight A. Bellinger, Robin A. Raymer, Stephanie McCorquodale, and Katherine Parker Ponder Neonatal or hepatocyte growth factor-potentiated adult gene therapy with a retroviral vector results in therapeutic levels of canine factor IX for hemophilia B, Blood, May 2003; 101: 3924 - 3932.
8 Rita Sarkar, Renee Tetreault, Guangping Gao, Lili Wang, Peter Bell, Randy Chandler, James M. Wilson, and Haig H. Kazazian, Jr Total correction of hemophilia A mice with canine FVIII using an AAV 8 serotype Blood, Feb 2004; 103: 1253 -1260.
9 Basu, U. Banerjee, S. An engineered EBV vector expressing human factor VIII and von Willebrand factor in cultured B-cells. J Gene Med. 2004 Jul;6(7):760-8
10 Valder R. Arruda, Hansell H. Stedman, Timothy C. Nichols, Mark E. Haskins, Matthew Nicholson, Roland W. Herzog, Linda B. Couto, and Katherine A. High Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model Blood, May 2005; 105: 3458 - 3464.
11 Juan Fang, Kairbaan Hodivala-Dilke, Bryon D. Johnson, Lily M. Du, Richard O. Hynes, Gilbert C. White, II, and David A. Wilcox Therapeutic expression of the platelet-specific integrin, aIIbB3 in a murine model for Glanzmann thrombasthenia Blood, Oct 2005; 106: 2671 - 2679.
12 Lingfei Xu, Timothy C. Nichols, Rita Sarkar, Stephanie McCorquodale, Dwight A. Bellinger, and Katherine P. Ponder Absence of a desmopressin response after therapeutic expression of factor VIII in hemophilia A dogs with liver-directed neonatal gene therapy PNAS 2005 102: 6080-6085; published online before print as 10.1073/pnas.0409249102
13 Paris Margaritis, Valder R. Arruda, Majed Aljamali, Rodney M. Camire, Alexander Schlachterman, and Katherine A. High Novel therapeutic approach for hemophilia using gene delivery of an engineered secreted activated Factor VII J. Clin. Invest., Apr 2004; 113: 1025 - 1031
14 Qizhen Shi, David A. Wilcox, Scot A. Fahs, Hartmut Weiler, Clive W. Wells, Brian C. Cooley, Drashti Desai, Patricia A. Morateck, Jack Gorski, and Robert R. Montgomery Factor VIII ectopically targeted to platelets is therapeutic in hemophilia A with high-titer inhibitory antibodies J. Clin. Invest., Jul 2006; 116: 1974 - 1982.
15 Margaritis P, High, KA Advances in gene therapy using factor VIIa in hemophilia. Seminars in Hematology. 2006 Jan (1Supp 1) : S101-4
16 Jianping Wen , Andrew GÅ\mez Vargas , Frederick A. Ofosu , Gonzalo Hortelano Sustained and therapeutic levels of human factor IX in hemophilia B mice implanted with microcapsules: key role of encapsulated cells, The Journal of Gene Medicine 2006 Mar;8(3):362-9
17 Bigger BW, Siapati EK, Mistry A, Waddington SN, Nivsarkar MS, Jacobs L, Perrett R, Holder MV, Ridler C, Kemball-Cook G, Ali RR, Forbes SJ, Coutelle C, Wright N, Alison M, Thrasher AJ, Bonnet D, Themis M. Permanent partial phenotypic correction and tolerance in a mouse model of hemophilia B by stem cell gene delivery of human factor IX. Gene Ther. 2006 Jan;13(2):117-26.
18 Jiang H, Pierce GF, Ozelo MC, de Paula EV, Vargas JA, Smith P, Sommer J, Luk A, Manno CS, High KA, Arruda VR. Evidence of multiyear factor IX expression by AAV-mediated gene transfer to skeletal muscle in an individual with severe hemophilia B. Mol Ther. 2006 Sep;14(3):452-5. Epub 2006 Jul 5.
19 Matsui H, Shibata M, Brown B, Labelle A, Hegadorn C, Andrews C, Hebbel RP, Galipeau J, Hough C, Lillicrap D. Ex Vivo Gene Therapy for Hemophilia A That Enhances Safe Delivery and Sustained In Vivo FVIII Expression From Lentivirally-engineered Endothelial Progenitors. Stem Cells. 2007 Jul 5; [Epub ahead of print]
20 Park SW, Choi SY. Long-Term Expression of von Willebrand Factor by a VSV-G Pseudotyped Lentivirus Enhances the Functional Activity of Secreted B-Domain-deleted Coagulation Factor VIII. Mol Cells. 2007 Aug 31;24(1):125-31
21 Brian D Brown, Alessio Cantore, Andrea Annoni, Lucia Sergi Sergi, Angelo Lombardo, Patrizia Della Valle, Armando D'Angelo, and Luigi Naldini A microRNA-regulated lentiviral vector mediates stable correction of hemophilia B mice Blood, Aug 2007; doi:10.1182/blood-2007-03-078493
Hemophilia Research1965 Cryoprecipitate used to treat Hemophilia.1
1968 First FIX and FVIII Concentrates.1
1982 FIX Gene cloned.1
1982 FVIII Gene cloned.1
1985 First viral inactivated factor concentrates become available.1
1992 First Recombinant product becomes available.1
1992 First Gene Therapy trials in mice.1
1993-1994 FIX First Gene Therapy in dogs.1
1993 FIX First Gene Therapy in humans 2% factor level increase.
1994 FIX Gene Therapy in dogs up to 300% increase for 1-2 months.2
1995-1996 First FIX Gene therapy in mice and dogs.1
1996 FVIII Gene Therapy in dogs well over therapeutic level for 1-2 weeks.31997 First Recombinant FIX product.1
1998 FVIII Gene Therapy in humans 4% factor level increase.1
1999 FIX Gene Therapy in humans B 1.6% increase.1
1999 FVIII Gene Therapy in mice produce 20 % increase for 11 months.4
2000 FVIII Gene Therapy in mice 4%-20% increase.5
2001 FIX Gene Therapy in humans 2%- 12% increase.1
2001 FVIII Gene Therapy in humans up to 3% increase.1
2002 FVIII Gene Therapy in mice up to 20 % increase.6
2003 FIX Gene Therapy in neonatal mice 150%-280% for more than 14 Months.72003 FIX Gene Therapy in neonatal dogs 12%- 36% increase for more than 14 Months.7
2004 FVIII Gene Therapy in mice produces 100% correction.8
2004 Vwb Factor Genetically produced in culture.9
2004 FIX Gene therapy in dogs 4%-14% increase for 3 years, observation ongoing.10
2005 Glannzman's Thrombasthenia Gene Therapy produces therapeutic levels in mice.11
2005 FVIII Gene Therapy in newborn mice and dogs produces 115% - 139% factor levels for 1 1/2 years, observation ongoing.12
2006 Gene Therapy produces FVIII stored in platelets to overcome inhibitors.14
2006 FVIIA Gene Therapy in mice produces therapeutic levels to overcome inhibitors.15
2006 FIX produced from microcapsule implanted in mice.16
2006 FIX Gene Therapy in mice produces Long Term Therapeutic levels.17
2007 FIX Gene Therapy in humans produces increases for over 3.7 years observation ongoing.18
2007 FVIII Gene Therapy in dogs and mice produces factor level increases 2%-4.5% for 27 weeks.19
2007 Vwb Factor produced by Gene Therapy.20
2007 FIX Gene Therapy in mice produces over 10% for more than 280 Days.21
2006 FVIIa Gene Therapy in mice produces FVIIa.13
1 C. Hough, D. Lillicrap (2005) Gene therapy for hemophilia: an imperative to succeed, Journal of Thrombosis and Haemostasis 3 (6), 1195-1205. doi:10.1111/j.1538-7836.2005.01401.x
2 Kay MA, Landen CN, Rothenberg SR, Taylor LA, Leland F, Wiehle S, Fang B, Bellinger D, Finegold M, Thompson AR, et al. In vivo hepatic gene therapy: complete albeit transient correction of factor IX deficiency in hemophilia B dogs.1: Proc Natl Acad Sci U S A. 1994 Mar 15;91(6):2353-7.
3 S Connelly, J Mount, A Mauser, JM Gardner, M Kaleko, A McClelland, and CD Jr Lothrop Complete short-term correction of canine hemophilia A by in vivo gene therapy Blood, Nov 1996; 88: 3846 - 3853.
4 Lang, H Study points to possible cure for hemophilia UNC-CH School of Medicine News Services June 9, 1999--No.369
5 Steven S. Fakharzadeh, Yue Zhang, Rita Sarkar, and Haig H. Kazazian, JrCorrection of the coagulation defect in hemophilia A mice through factor VIII expression in skin Blood, May 2000; 95: 2799 - 2805.
6 Arnette R. New gene therapy treatment works in mice with hemophilia A. UNC School of Medicine News Services June 10, 2002 -- No. 332
7 Lingfei Xu, Cuihua Gao, Mark S. Sands, Shi-Rong Cai, Timothy C. Nichols, Dwight A. Bellinger, Robin A. Raymer, Stephanie McCorquodale, and Katherine Parker Ponder Neonatal or hepatocyte growth factor-potentiated adult gene therapy with a retroviral vector results in therapeutic levels of canine factor IX for hemophilia B, Blood, May 2003; 101: 3924 - 3932.
8 Rita Sarkar, Renee Tetreault, Guangping Gao, Lili Wang, Peter Bell, Randy Chandler, James M. Wilson, and Haig H. Kazazian, Jr Total correction of hemophilia A mice with canine FVIII using an AAV 8 serotype Blood, Feb 2004; 103: 1253 -1260.
9 Basu, U. Banerjee, S. An engineered EBV vector expressing human factor VIII and von Willebrand factor in cultured B-cells. J Gene Med. 2004 Jul;6(7):760-8
10 Valder R. Arruda, Hansell H. Stedman, Timothy C. Nichols, Mark E. Haskins, Matthew Nicholson, Roland W. Herzog, Linda B. Couto, and Katherine A. High Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model Blood, May 2005; 105: 3458 - 3464.
11 Juan Fang, Kairbaan Hodivala-Dilke, Bryon D. Johnson, Lily M. Du, Richard O. Hynes, Gilbert C. White, II, and David A. Wilcox Therapeutic expression of the platelet-specific integrin, aIIbB3 in a murine model for Glanzmann thrombasthenia Blood, Oct 2005; 106: 2671 - 2679.
12 Lingfei Xu, Timothy C. Nichols, Rita Sarkar, Stephanie McCorquodale, Dwight A. Bellinger, and Katherine P. Ponder Absence of a desmopressin response after therapeutic expression of factor VIII in hemophilia A dogs with liver-directed neonatal gene therapy PNAS 2005 102: 6080-6085; published online before print as 10.1073/pnas.0409249102
13 Paris Margaritis, Valder R. Arruda, Majed Aljamali, Rodney M. Camire, Alexander Schlachterman, and Katherine A. High Novel therapeutic approach for hemophilia using gene delivery of an engineered secreted activated Factor VII J. Clin. Invest., Apr 2004; 113: 1025 - 1031
14 Qizhen Shi, David A. Wilcox, Scot A. Fahs, Hartmut Weiler, Clive W. Wells, Brian C. Cooley, Drashti Desai, Patricia A. Morateck, Jack Gorski, and Robert R. Montgomery Factor VIII ectopically targeted to platelets is therapeutic in hemophilia A with high-titer inhibitory antibodies J. Clin. Invest., Jul 2006; 116: 1974 - 1982.
15 Margaritis P, High, KA Advances in gene therapy using factor VIIa in hemophilia. Seminars in Hematology. 2006 Jan (1Supp 1) : S101-4
16 Jianping Wen , Andrew GÅ\mez Vargas , Frederick A. Ofosu , Gonzalo Hortelano Sustained and therapeutic levels of human factor IX in hemophilia B mice implanted with microcapsules: key role of encapsulated cells, The Journal of Gene Medicine 2006 Mar;8(3):362-9
17 Bigger BW, Siapati EK, Mistry A, Waddington SN, Nivsarkar MS, Jacobs L, Perrett R, Holder MV, Ridler C, Kemball-Cook G, Ali RR, Forbes SJ, Coutelle C, Wright N, Alison M, Thrasher AJ, Bonnet D, Themis M. Permanent partial phenotypic correction and tolerance in a mouse model of hemophilia B by stem cell gene delivery of human factor IX. Gene Ther. 2006 Jan;13(2):117-26.
18 Jiang H, Pierce GF, Ozelo MC, de Paula EV, Vargas JA, Smith P, Sommer J, Luk A, Manno CS, High KA, Arruda VR. Evidence of multiyear factor IX expression by AAV-mediated gene transfer to skeletal muscle in an individual with severe hemophilia B. Mol Ther. 2006 Sep;14(3):452-5. Epub 2006 Jul 5.
19 Matsui H, Shibata M, Brown B, Labelle A, Hegadorn C, Andrews C, Hebbel RP, Galipeau J, Hough C, Lillicrap D. Ex Vivo Gene Therapy for Hemophilia A That Enhances Safe Delivery and Sustained In Vivo FVIII Expression From Lentivirally-engineered Endothelial Progenitors. Stem Cells. 2007 Jul 5; [Epub ahead of print]
20 Park SW, Choi SY. Long-Term Expression of von Willebrand Factor by a VSV-G Pseudotyped Lentivirus Enhances the Functional Activity of Secreted B-Domain-deleted Coagulation Factor VIII. Mol Cells. 2007 Aug 31;24(1):125-31
21 Brian D Brown, Alessio Cantore, Andrea Annoni, Lucia Sergi Sergi, Angelo Lombardo, Patrizia Della Valle, Armando D'Angelo, and Luigi Naldini A microRNA-regulated lentiviral vector mediates stable correction of hemophilia B mice Blood, Aug 2007; doi:10.1182/blood-2007-03-078493
Hemophilia A affects about 15,000 in the U.S.