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Haemophilia

Vol. 515 No. 7528_supp ppS157-S200

In this Supplement
Hopes are rising rapidly for people with the bleeding disorder haemophilia. The advent of longer-lasting blood-clotting factors is making treatment less onerous, and — on the horizon — gene therapy offers a potential cure.

Cover Art: Jessica Fortner

Outlook

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Haemophilia
- Herb Brody
Nature 515, S157 (27 November 2014)
Born in the blood
- Neil Savage
Nature 515, S158-S159 (27 November 2014)
Gene therapy: Genie in a vector
- Julie Gould
Nature 515, S160–S161 (27 November 2014)
Clotting factors: Stretching time
- Neil Savage
Nature 515, S162-S164 (27 November 2014)
Perspective: The fix is in
- Stephen Pemberton
Nature 515, S165 (27 November 2014)
Immunology: Oral solutions
- Elie Dolgin
Nature 515, S166-S167 (27 November 2014)
Thrombosis: Balancing act
- Cassandra Willyard
Nature 515, S168-S169 (27 November 2014)
Orthopaedics: Joint effort
- Katharine Gammon
Nature 515, S170-S171 (27 November 2014)
Animal models: Dogged pursuit
- Emily Sohn
Nature 515, S172-173 (27 November 2014)

Video

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Rockstars and flying cars

Zak is 13 and he has haemophilia. His daily routine is a bit different from that of most teenagers. Whereas other children might just need to brush their teeth in the morning, every day Zak has to give himself an injection. In this video, Zak and his family give an insight into what living with haemophilia is really like — the ups and the downs, the misconceptions and the realities. We also hear about the options that might be available in the future for Zak and others with haemophilia if cutting-edge research from University College London leads to a cure.

Collection

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Intron 22 homologous regions are implicated in exons 1–22 duplications of the F8 gene
- Nathalie Lannoy
- Bernard Grisart
- Stéphane Eeckhoudt
- Christine Verellen-Dumoulin
- Catherine Lambert
- Miikka Vikkula
- Cédric Hermans
European Journal of Human Genetics 21, 970–976 (09 January 2013)
Integration-deficient Lentiviral Vectors Expressing Codon-optimized R338L Human FIX Restore Normal Hemostasis in Hemophilia B Mice
- Thipparat Suwanmanee
- Genlin Hu
- Tong Gui
- Cynthia C Bartholomae
- Ina Kutschera
- Christof von Kalle
- Manfred Schmidt
- Paul E Monahan
- Tal Kafri
Molecular Therapy 22, 567–574 (2014)
In vivo genome editing restores haemostasis in a mouse model of haemophilia
- Hojun Li
- Virginia Haurigot
- Yannick Doyon
- Tianjian Li
- Sunnie Y. Wong
- Anand S. Bhagwat
- Nirav Malani
- Xavier M. Anguela
- Rajiv Sharma
- Lacramiora Ivanciu
- Samuel L. Murphy
- Jonathan D. Finn
- Fayaz R. Khazi
- Shangzhen Zhou
- David E. Paschon
- Edward J. Rebar
- Frederic D. Bushman
- Philip D. Gregory
- Michael C. Holmes
- Katherine A. High
Nature 475, 217–221 (26 June 2011)
A bispecific antibody to factors IXa and X restores factor VIII hemostatic activity in a hemophilia A model
- Takehisa Kitazawa
- Tomoyuki Igawa
- Zenjiro Sampei
- Atsushi Muto
- Tetsuo Kojima
- Tetsuhiro Soeda
- Kazutaka Yoshihashi
- Yukiko Okuyama-Nishida
- Hiroyuki Saito
- Hiroyuki Tsunoda
- Tsukasa Suzuki
- Hideki Adachi
- Taro Miyazaki
- Shinya Ishii
- Mika Kamata-Sakurai
- Takeo Iida
- Aya Harada
- Keiko Esaki
- Miho Funaki
- Chifumi Moriyama
- Eriko Tanaka
- Yasufumi Kikuchi
- Tetsuya Wakabayashi
- Manabu Wada
- Masaaki Goto
- Takeshi Toyoda
- Atsunori Ueyama
- Sachiyo Suzuki
- Kenta Haraya
- Tatsuhiko Tachibana
- Yoshiki Kawabe
- Midori Shima
- Akira Yoshioka
- Kunihiro Hattori
Nature Medicine 18, 1570–1574 (30 September 2012)
A complex substitute: antibody therapy for hemophilia
- David Lillicrap
Nature Medicine 18, 1460–1461 (05 October 2012)

more online

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For more on haematological diseases from nature.com, see:
nature.com/subjects/haematological-diseases

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Supplement

Haemophilia

In this Supplement

Outlook

Haemophilia

Born in the blood

Gene therapy: Genie in a vector

Clotting factors: Stretching time

Perspective: The fix is in

Immunology: Oral solutions

Thrombosis: Balancing act

Orthopaedics: Joint effort

Animal models: Dogged pursuit

Video

Rockstars and flying cars

Collection

Intron 22 homologous regions are implicated in exons 1–22 duplications of the F8 gene

Integration-deficient Lentiviral Vectors Expressing Codon-optimized R338L Human FIX Restore Normal Hemostasis in Hemophilia B Mice

In vivo genome editing restores haemostasis in a mouse model of haemophilia

A bispecific antibody to factors IXa and X restores factor VIII hemostatic activity in a hemophilia A model

A complex substitute: antibody therapy for hemophilia

more online

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