Riding the metal wave: A review of the latest developments in metal-based anticancer agents
Metal-based complexes have demonstrated significant anticancer effects and have emerged
as candidates with great potential for cancer therapy. These agents are effective in a variety …
as candidates with great potential for cancer therapy. These agents are effective in a variety …
Rationale and strategies for the development of safe and effective optimized AAV vectors for human gene therapy
A Srivastava - Molecular Therapy-Nucleic Acids, 2023 - cell.com
Recombinant adeno-associated virus (AAV) vectors have been, or are currently in use, in
332 phase I/II/III clinical trials in a number of human diseases, and in some cases …
332 phase I/II/III clinical trials in a number of human diseases, and in some cases …
Gene therapy with Fidanacogene Elaparvovec in adults with hemophilia B
A Cuker, K Kavakli, L Frenzel, JD Wang… - … England Journal of …, 2024 - Mass Medical Soc
Background Fidanacogene elaparvovec, an adeno-associated virus (AAV) gene-therapy
vector for hemophilia B containing a high-activity human factor IX variant (FIX-R338L/FIX …
vector for hemophilia B containing a high-activity human factor IX variant (FIX-R338L/FIX …
[HTML][HTML] Adeno-associated viruses for gene therapy–clinical implications and liver-related complications, a guide for hepatologists
MM Mücke, S Fong, GR Foster, D Lillicrap… - Journal of …, 2024 - Elsevier
Gene therapy has garnered increasing interest over recent decades. Several therapies
employing gene transfer mechanisms have been developed, and, of these, adeno …
employing gene transfer mechanisms have been developed, and, of these, adeno …
[PDF][PDF] Molecular evaluation and vector integration analysis of HCC complicating AAV gene therapy for hemophilia B
M Schmidt, GR Foster, M Coppens, H Thomsen… - Blood advances, 2023 - Elsevier
COI notes: Manfred Schmidt was CEO and founder of GeneWerk (Protagene CGT) GmbH.
Graham R. Foster has received consultancy and speaker fees from Abbvie, Biomarin …
Graham R. Foster has received consultancy and speaker fees from Abbvie, Biomarin …
Hemophilia gene therapy: the end of the beginning?
D De Wolf, K Singh, MK Chuah… - Human Gene …, 2023 - liebertpub.com
Extensive preclinical research over the past 30 years has culminated in the recent regulatory
approval of several gene therapy products for hemophilia. Based on the efficacy and safety …
approval of several gene therapy products for hemophilia. Based on the efficacy and safety …
[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 …
Gene therapy approaches for the treatment of hemophilia B
AB Soroka, SG Feoktistova, ON Mityaeva… - International Journal of …, 2023 - mdpi.com
In contrast to the standard enzyme-replacement therapy, administered from once per 7–14
days to 2–3 times a week in patients with severe hemophilia B, as a result of a single …
days to 2–3 times a week in patients with severe hemophilia B, as a result of a single …
Evaluating gene therapy as a potential paradigm shift in treating severe hemophilia
CD Thornburg, DH Simmons, A von Drygalski - BioDrugs, 2023 - Springer
Hemophilia is characterized by a deficiency in coagulation factors VIII or IX. The general
standard of care for severe hemophilia is frequent intravenous recombinant or plasma …
standard of care for severe hemophilia is frequent intravenous recombinant or plasma …
Adeno-associated viral vector integration: implications for long-term efficacy and safety
P Batty, D Lillicrap - Journal of Thrombosis and Haemostasis, 2024 - Elsevier
Adeno-associated viral vector (AAV) gene therapy provides a promising platform for
treatment of monogenic inherited disorders. Clinical studies have demonstrated long-term …
treatment of monogenic inherited disorders. Clinical studies have demonstrated long-term …