Severe Combined Immunodeficiency (SCID) News and Research

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Using novel gene therapy to cure infants born with severe combined immunodeficiency

Using novel gene therapy to cure infants born with severe combined immunodeficiency

Study to determine lowest dose of chemotherapy needed for babies with SCID

Study to determine lowest dose of chemotherapy needed for babies with SCID

IQWiG assesses benefit of newborn screening for severe combined immunodeficiency

IQWiG assesses benefit of newborn screening for severe combined immunodeficiency

Genome engineering-based methods pave way for new treatment of patients with sickle cell disease

Genome engineering-based methods pave way for new treatment of patients with sickle cell disease

Gene therapy can restore immune systems of children and young adults with SCID-X1

Gene therapy can restore immune systems of children and young adults with SCID-X1

Experimental gene therapy may improve health outcomes for patients with some forms of blood disorders

Experimental gene therapy may improve health outcomes for patients with some forms of blood disorders

Finding could lead to more effective, less invasive treatment for 'bubble boy' disease

Finding could lead to more effective, less invasive treatment for 'bubble boy' disease

Newborn screening for SCID holds promise that affected children can lead healthy lives

Newborn screening for SCID holds promise that affected children can lead healthy lives

Study examines national impact of newborn screening test for SCID

Study examines national impact of newborn screening test for SCID

Newborn screening indicates that incidence of SCID is higher than previously believed

Newborn screening indicates that incidence of SCID is higher than previously believed

Blood-forming stem cell transplantation benefits infants with severe combined immunodeficiency

Blood-forming stem cell transplantation benefits infants with severe combined immunodeficiency

Two pediatricians win March of Dimes/Col. Harland D. Sanders Lifetime Achievement Award in Genetics

Two pediatricians win March of Dimes/Col. Harland D. Sanders Lifetime Achievement Award in Genetics

Genetically engineered cell therapies demonstrate early efficacy, safety in patients with blood disorders

Genetically engineered cell therapies demonstrate early efficacy, safety in patients with blood disorders

UCSF to receive grant to study gene sequencing aimed at detecting disorders in newborns

UCSF to receive grant to study gene sequencing aimed at detecting disorders in newborns

Orphan, St George's sign licensing agreement to develop new therapies for metabolic disorders

Orphan, St George's sign licensing agreement to develop new therapies for metabolic disorders

Refinements in gene therapy prove beneficial for ‘bubble babies’

Refinements in gene therapy prove beneficial for ‘bubble babies’

Gene therapy regimen can safely restore immune systems to children with Bubble Boy disease

Gene therapy regimen can safely restore immune systems to children with Bubble Boy disease

FTY720 improves locomotor recovery in mice with SCI

FTY720 improves locomotor recovery in mice with SCI

Severe combined immunodeficiency treated successfully with gene therapy

Severe combined immunodeficiency treated successfully with gene therapy

Researchers find temperature-sensing protein in immune cells

Researchers find temperature-sensing protein in immune cells