Lentiviral hematopoietic stem cell gene therapy in inherited metabolic disorders
Human Gene Therapy , Volume 25 - Issue 10 p. 862- 865
After more than 20 years of development, lentiviral hematopoietic stem cell gene therapy has entered the stage of initial clinical implementation for immune deficiencies and storage disorders. This brief review summarizes the development and applications, focusing on the lysosomal enzyme deficiencies, especially Pompe disease.
|Human Gene Therapy|
|Organisation||Erasmus University Rotterdam|
Wagemaker, G. (2014). Lentiviral hematopoietic stem cell gene therapy in inherited metabolic disorders. Human Gene Therapy (Vol. 25, pp. 862–865). doi:10.1089/hum.2014.102