Update on clinical gene therapy for hemophilia
GQ Perrin, RW Herzog… - Blood, The Journal of the …, 2019 - ashpublications.org
In contrast to other diverse therapies for the X-linked bleeding disorder hemophilia that are
currently in clinical development, gene therapy holds the promise of a lasting cure with a …
currently in clinical development, gene therapy holds the promise of a lasting cure with a …
Gene therapy for hemophilia: what does the future hold?
Recent phase I/II adeno-associated viral vector-mediated gene therapy clinical trials have
reported remarkable success in ameliorating disease phenotype in hemophilia A and B …
reported remarkable success in ameliorating disease phenotype in hemophilia A and B …
A molecular revolution in the treatment of hemophilia
For decades, the monogenetic bleeding disorders hemophilia A and B (coagulation factor
VIII and IX deficiency) have been treated with systemic protein replacement therapy. Now …
VIII and IX deficiency) have been treated with systemic protein replacement therapy. Now …
Clinical advances in gene therapy updates on clinical trials of gene therapy in haemophilia
F Peyvandi, I Garagiola - Haemophilia, 2019 - Wiley Online Library
Gene therapy is rapidly becoming a new therapeutic strategy for haemophilia A and B
treatment. In the 1990s, studies in animal models showed that adeno‐associated vectors …
treatment. In the 1990s, studies in animal models showed that adeno‐associated vectors …
[HTML][HTML] Deciphering conundrums of adeno-associated virus liver-directed gene therapy: focus on hemophilia
Adeno-associated virus gene therapy has been the subject of intensive investigation for
monogenic disease gene addition therapy for more than 25 years, yet few therapies have …
monogenic disease gene addition therapy for more than 25 years, yet few therapies have …
Protein-engineered coagulation factors for hemophilia gene therapy
Hemophilia A (HA) and hemophilia B (HB) are X-linked bleeding disorders due to
inheritable deficiencies in either coagulation factor VIII (FVIII) or factor IX (FIX), respectively …
inheritable deficiencies in either coagulation factor VIII (FVIII) or factor IX (FIX), respectively …
AAV gene therapy in companion dogs with severe hemophilia: Real-world long-term data on immunogenicity, efficacy, and quality of life
The hemophilias are the most common severe inherited bleeding disorders and are caused
by deficiency of clotting factor (F) VIII (hemophilia A) or FIX (hemophilia B). The resultant …
by deficiency of clotting factor (F) VIII (hemophilia A) or FIX (hemophilia B). The resultant …
Cellular stress and coagulation factor production: when more isn't necessarily better
Z Chen, RW Herzog, RJ Kaufman - Journal of Thrombosis and …, 2023 - Elsevier
Remarkably, it has been 40 years since the isolation of the two genes involved in
hemophilia A (HA) and hemophilia B (HB), encoding clotting factors VIII (FVIII) and IX (FIX) …
hemophilia A (HA) and hemophilia B (HB), encoding clotting factors VIII (FVIII) and IX (FIX) …
Activated protein C has a regulatory role in factor VIII function
AR Wilhelm, NA Parsons… - Blood, The Journal …, 2021 - ashpublications.org
Mechanisms thought to regulate activated factor VIII (FVIIIa) cofactor function include A2-
domain dissociation and activated protein C (APC) cleavage. Unlike A2-domain …
domain dissociation and activated protein C (APC) cleavage. Unlike A2-domain …
Analysis of vector genome integrations in multicentric lymphoma after AAV gene therapy in a severe hemophilia A dog
L Van Gorder, BS Doshi, E Willis, TC Nichols… - … Therapy Methods & …, 2023 - cell.com
Adeno-associated viral (AAV) vectors have traditionally been viewed as predominantly
nonintegrating, with limited concerns for oncogenesis. However, accumulating preclinical …
nonintegrating, with limited concerns for oncogenesis. However, accumulating preclinical …