X-linked Severe Combined Immunodeficiency
Last Posted: Dec 21, 2020
- Immune Reconstitution After Gene Therapy Approaches in Patients With X-Linked Severe Combined Immunodeficiency Disease.
Blanco Elena et al. Frontiers in immunology 2020 11608653
- Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1.
Mamcarz Ewelina et al. The New England journal of medicine 2019 Apr 380(16) 1525-1534
- Gene Therapy Approaches to Immunodeficiency.
Ghosh Sujal et al. Hematology/oncology clinics of North America 2017 Oct 31(5) 823-834
- Gene Therapy for X-Linked Severe Combined Immunodeficiency: Where Do We Stand?
Cavazzana Marina et al. Human gene therapy 2016 Feb (2) 108-16
- X-linked severe combined immunodeficiency
From NCATS Genetic and Rare Diseases Information Center
- Sib understanding of genetics and attitudes toward carrier testing for X-linked severe combined immunodeficiency.
Fanos J H, et al. American journal of medical genetics 2001 1 (1) 46-56
Rare Disease PHGKB is an online, continuously updated, searchable database of published scientific literature, CDC and NIH resources, and other information that address the public health impact and translation of genomic and other precision health discoveries into improved health outcomes related to rare diseases...more
Selected Rare Diseases
- Alpha-1 Antitrypsin Deficiency
- Amyotrophic Lateral Sclerosis
- Brugada Syndrome
- Cerebral Palsy
- Cystic Fibrosis
- Duchenne Muscular Dystrophy
- Erythema Multiforme
- Familial Mediterranean Fever
- Fragile X Syndrome
- Gaucher Disease
- Graves Disease
- Huntington Disease
- Myasthenia Gravis
- Retinitis Pigmentosa
- Severe Combined Immunodeficiency
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