X
Search Filters
Format Format
Subjects Subjects
Subjects Subjects
X
Sort by Item Count (A-Z)
Filter by Count
index medicus (29) 29
humans (25) 25
transplantation (18) 18
gene therapy (15) 15
male (15) 15
immunology (14) 14
children (11) 11
female (11) 11
child (10) 10
genetic therapy (9) 9
hematopoietic stem cells (9) 9
mutation (9) 9
infant (8) 8
patients (8) 8
pediatrics (8) 8
research (8) 8
stem cells (8) 8
stem-cell transplantation (8) 8
medicine, research & experimental (7) 7
severe combined immunodeficiency (7) 7
usage (7) 7
abridged index medicus (6) 6
adolescent (6) 6
care and treatment (6) 6
child, preschool (6) 6
expression (6) 6
genetic aspects (6) 6
hematology (6) 6
immunodeficiency (6) 6
adenosine deaminase - deficiency (5) 5
allergy and immunology (5) 5
analysis (5) 5
genetics & heredity (5) 5
hematopoietic stem cell transplantation (5) 5
lymphocytes (5) 5
reconstitution (5) 5
adenosine (4) 4
allergy (4) 4
article (4) 4
blood (4) 4
cell biology (4) 4
combined immune-deficiency (4) 4
gene-therapy (4) 4
genetic therapy - methods (4) 4
health aspects (4) 4
hematopoietic stem cells - metabolism (4) 4
hemopoiesis (4) 4
immunologic diseases. allergy (4) 4
outcomes (4) 4
severe combined immunodeficiency - therapy (4) 4
ada (3) 3
adenosine deaminase (3) 3
adult (3) 3
agammaglobulinemia - therapy (3) 3
age (3) 3
autoimmunity (3) 3
biotechnology & applied microbiology (3) 3
bone marrow (3) 3
bone-marrow (3) 3
bone-marrow-transplantation (3) 3
chronic granulomatous-disease (3) 3
clinical-features (3) 3
defects (3) 3
deficiency (3) 3
diagnosis (3) 3
efficacy (3) 3
experience (3) 3
follow-up studies (3) 3
gastroenterology & hepatology (3) 3
gene mutations (3) 3
genes (3) 3
genetic disorders (3) 3
genetics (3) 3
immune reconstitution (3) 3
immunology and allergy (3) 3
infections (3) 3
lentivirus (3) 3
leukodystrophy, metachromatic - therapy (3) 3
mouse model (3) 3
muscular dystrophy (3) 3
next generation sequencing (3) 3
pedigree (3) 3
phenotype (3) 3
phenotypes (3) 3
primary immunodeficiencies (3) 3
research article (3) 3
risk factors (3) 3
severe combined immunodeficiency - genetics (3) 3
studies (3) 3
t cells (3) 3
transplants & implants (3) 3
treatment outcome (3) 3
wasp (3) 3
wiskott-aldrich syndrome (3) 3
wiskott-aldrich syndrome - therapy (3) 3
wiskott-aldrich-syndrome (3) 3
actin (2) 2
adenosine deaminase - genetics (2) 2
adenosine deaminase - therapeutic use (2) 2
adenosine deaminase deficiency (2) 2
more...
Language Language
Publication Date Publication Date
Click on a bar to filter by decade
Slide to change publication date range


Lancet, The, ISSN 0140-6736, 2016, Volume 388, Issue 10043, pp. 476 - 487
Journal Article
Science, ISSN 0036-8075, 8/2013, Volume 341, Issue 6148, pp. 865 - 865
Journal Article
Science, ISSN 0036-8075, 8/2013, Volume 341, Issue 6148, pp. 864 - 864
Journal Article
Journal Article
EMBO Molecular Medicine, ISSN 1757-4676, 12/2015, Volume 7, Issue 12, pp. 1513 - 1528
Journal Article
Journal Article
Journal Article
Blood, ISSN 0006-4971, 07/2016, Volume 128, Issue 1, pp. 45 - 54
Journal Article
The Journal of Allergy and Clinical Immunology, ISSN 0091-6749, 09/2019, Volume 144, Issue 3, pp. 825 - 838
Thrombocytopenia is a serious issue for all patients with classical Wiskott-Aldrich syndrome (WAS) and X-linked thrombocytopenia (XLT) because it causes severe... 
Wiskott-Aldrich syndrome | X-linked thrombocytopenia | gene therapy | platelets | CLEARANCE | CD40 LIGAND | CELL TRANSPLANTATION | SYNDROME PROTEIN | NEUTROPHILS | SPLENECTOMY | IMMUNOLOGY | ACTIVATED PLATELETS | ALLERGY | MUTATIONS | EXPRESSION | Genetic research | Care and treatment | Genetic aspects | Gene therapy | Analysis | Flow cytometry | Pediatrics | Transplants & implants | Medical services | Clinical trials | Agglomeration | Coexistence | Bleeding | Defects | Proteins | Genotype & phenotype | Signal transduction | Protein composition | Restoration | Blood platelets | Ultrastructure | Thrombocytopenia | Phenotypes | Immune reconstitution | Electron microscopy | Patients | Microscopy | Protein expression | Software | Mutation | Platelets | FERMT3, Fermitin family homolog 3 | TEM, Transmission electron microscopy | WAS, Wiskott-Aldrich syndrome | HMGB1, High-mobility group box 1 | δ-g, Electron-dense granule | GPX1, Glutathione peroxidase 1 | FU, Follow-up | sCD62P, Soluble P-selectin | sCD40L, Soluble CD40 ligand | BAFF, B cell–activating factor | CD62P, P-selectin | HSCT, Hematopoietic stem cell transplantation | WASp, Wiskott-Aldrich syndrome protein | MFI, Mean fluorescence intensity | LV, Lentivirus | CT, Closure time | GT, Gene therapy | OCS, Open canalicular system | PRP, Platelet-rich plasma | vWF, von Willebrand factor | STAT3, Signal transducer and activator of transcription 3 | HD, Healthy donor | ADP, Adenosine diphosphate | ROS, Reactive oxygen species | XLT, X-linked thrombocytopenia
Journal Article
Molecular Therapy, ISSN 1525-0016, 03/2018, Volume 26, Issue 3, pp. 917 - 931
Loss of adenosine deaminase activity leads to severe combined immunodeficiency (ADA-SCID); production and function of T, B, and natural killer (NK) cells are... 
immune deficiency | retroviral vector | safety | adenosine deaminase | gene therapy | immune reconstitution | ADA-SCID | opportunistic infection | MEDICINE, RESEARCH & EXPERIMENTAL