Future of genetic therapies for rare genetic diseases: what to expect for the next 15 years?

LAM Braga, CG Conte Filho… - Therapeutic Advances in …, 2022 - journals.sagepub.com
Introduction: Rare genetic diseases affect millions of people worldwide. Most of them are
caused by defective genes that impair quality of life and can lead to premature death. As …

Gene Editing as an Alternative to Retroviral Vectors for Wiskott-Aldrich syndrome Gene therapy

A Gutiérrez-Guerrero - 2018 - digibug.ugr.es
One of the safest and most efficient treatments for monogenic diseases affecting the
hematopoietic system is gene therapy using lentiviral vectors (LVs) and gammaretroviral …

Gene editing as an alternative to retroviral vectors for wiskott-aldrich syndrome gene therapy

MAG Guerrero - 2017 - dialnet.unirioja.es
One of the safest and most efficient treatments for monogenic diseases affecting the
hematopoietic system is gene therapy using lentiviral vectors (LVs) and gammaretroviral …