Department of Biotechnology
inStem (Institute for Stem Cell Science and Regenerative Medicine)

Haemophilia gene therapy-Update on new country initiatives.

Publication Type

Journal Article

Date of Publication

May 1, 2022

Journal

Haemophilia : the official journal of the World Federation of Hemophilia

Volume/Issue

28 Suppl 4

ISSN

1365-2516

Gene therapy is emerging as a potential cure for haemophilia. Gene therapy is a one-time treatment that can elevate factor levels for many years and minimize or eliminate the need for clotting factor concentrate (CFC) replacement therapy. However, there is a paucity of reports on gene therapy efforts in countries outside of North America or Europe, especially in low-and-middle-income countries (LMIC). All indications are that gene therapy will be one of standard care treatments for haemophilia in the future. Still, it may not be accessible to many countries due to various barriers and challenges. At the same time, each country may formulate solutions that may be used globally.

Alternate Journal

Haemophilia

PubMed ID

35521726

Authors

Ulrike M Reiss
Johnny Mahlangu
Tsukasa Ohmori
Margareth C Ozelo
Alok Srivastava
Lei Zhang

Keywords

Hemophilia A
Brazil
Developing Countries
Europe
Humans
Genetic Therapy
Dependovirus