Since its discovery as a genome editing tool, the clustered regularly interspaced short
palindromic repeats and CRISPR-associated protein 9 (CRISPR/Cas9) system has opened …

Leukodystrophies are a heterogenous group of inherited, degenerative encephalopathies,
that if left untreated, are often lethal at an early age. Although some of the leukodystrophies …

X-linked severe combined immunodeficiency (X-SCID) is a primary immunodeficiency that is
caused by mutations in the interleukin-2 receptor gamma (IL2RG) gene. Some patients …

Sulfatases are enzymes that catalyze the removal of sulfate from biological substances, an
essential process for the homeostasis of the body. They are commonly activated by the …

Advancements in gene delivery and editing have expanded the applications of autologous
hematopoietic stem and progenitor cells (HSPCs) for the treatment of monogenic and …

Lessons learned from decades-long practice in the transplantation of hematopoietic stem
and progenitor cells (HSPCs) to treat severe inherited disorders or cancer, have set the …

Chinese hamster ovary (CHO) cells are the commonly used mammalian host system to
manufacture recombinant proteins including monoclonal antibodies. However unfavorable …

Metachromatic leukodystrophy (MLD) is a neurodegenerative lysosomal storage disorder
caused by biallelic pathogenic variants in the gene encoding arylsulfatase A. Disease onset …

Metachromatic leukodystrophy (MLD) is a rare leukoencephalopathy caused by pathogenic
mutations in the ARSA gene. It manifests as severe motor symptoms, mental problems, and …

X-linked severe combined immunodeficiency (X-SCID) is a rare primary immunodeficiency
disorder with X-chromosome-linked recessive inheritance, that is caused by the mutations of …