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Journal Article
Blood, ISSN 0006-4971, 05/2016, Volume 127, Issue 21, pp. 2536 - 2545
Journal Article
Biology of Blood and Marrow Transplantation, ISSN 1083-8791, 2016, Volume 22, Issue 10, pp. 1851 - 1860
Journal Article
Seminars in Fetal and Neonatal Medicine, ISSN 1744-165X, 2015, Volume 21, Issue 1, pp. 57 - 65
Summary The inherited bone marrow failure syndromes (IBMFS) are a rare yet clinically important cause of neonatal hematological and non-hematological... 
Neonatal and Perinatal Medicine | Dyskeratosis congenita | Neonate | Bone marrow failure | Fanconi anemia | Cytopenia | Cancer | ABSENT RADIUS SYNDROME | AMEGAKARYOCYTIC THROMBOCYTOPENIA | SEVERE CONGENITAL NEUTROPENIA | ACUTE MYELOGENOUS LEUKEMIA | G-CSF THERAPY | FANCONI-ANEMIA | CLINICAL PRESENTATION | DYSKERATOSIS-CONGENITA | DIAMOND-BLACKFAN ANEMIA | MOLECULAR PATHOGENESIS | PEDIATRICS | Bone Marrow Diseases - diagnosis | Radius | Hemoglobinuria, Paroxysmal - congenital | Humans | Anemia, Aplastic | Exocrine Pancreatic Insufficiency - diagnosis | Lipomatosis - therapy | Neutropenia - congenital | Thrombocytopenia - complications | Neutropenia - complications | Upper Extremity Deformities, Congenital - therapy | Neutropenia - diagnosis | Dyskeratosis Congenita - complications | Bone Marrow Diseases - therapy | Dyskeratosis Congenita - diagnosis | Dyskeratosis Congenita - therapy | Anemia, Diamond-Blackfan - therapy | Thrombocytopenia - therapy | Anemia, Diamond-Blackfan - complications | Hemoglobinuria, Paroxysmal - diagnosis | Infant, Newborn | Bone Marrow Diseases - complications | Exocrine Pancreatic Insufficiency - complications | Fanconi Anemia - complications | Upper Extremity Deformities, Congenital - complications | Anemia, Diamond-Blackfan - diagnosis | Upper Extremity Deformities, Congenital - diagnosis | Fanconi Anemia - diagnosis | Exocrine Pancreatic Insufficiency - therapy | Lipomatosis - diagnosis | Neutropenia - therapy | Lipomatosis - complications | Thrombocytopenia - diagnosis | Fanconi Anemia - therapy | Hemoglobinuria, Paroxysmal - therapy | Hemoglobinuria, Paroxysmal - complications | Infants (Newborn) | Genetic aspects | Health aspects | Epidemiology | Fanconi's anemia
Journal Article
Annals of Neurology, ISSN 0364-5134, 02/2017, Volume 81, Issue 2, pp. 212 - 226
ObjectivesFriedreich's ataxia is a devastating neurological disease currently lacking any proven treatment. We studied the neuroprotective effects of the... 
TRIPLET-REPEAT EXPANSION | SIGNALING PATHWAYS | COLONY-STIMULATING FACTOR | ADULT NEUROGENESIS | FRATAXIN DEFICIENCY LEADS | TAIL NERVES | STEM-CELL FACTOR | OPEN-LABEL | AMYOTROPHIC-LATERAL-SCLEROSIS | G-CSF | NEUROSCIENCES | CLINICAL NEUROLOGY | Friedreich Ataxia - metabolism | Friedreich Ataxia - drug therapy | Humans | Mice, Inbred C57BL | Mice, Transgenic | Peripheral Nerves - drug effects | Iron-Binding Proteins - metabolism | Stem Cell Factor - administration & dosage | Friedreich Ataxia - physiopathology | Granulocyte Colony-Stimulating Factor - pharmacology | Animals | Neuroprotective Agents - pharmacology | Stem Cell Factor - pharmacology | Behavior, Animal - drug effects | Mice | Neural Conduction - drug effects | Neuroprotective Agents - administration & dosage | Disease Models, Animal | Granulocyte Colony-Stimulating Factor - administration & dosage | Ataxia | Cytokines | Rodents | Stem cells | Cerebellum | Neuroprotection | Conduction | Spinal cord | Regulators | Performance tests | Animal models | Pathogenesis | mRNA | Cerebrospinal fluid | Motor task performance | Stem cell factor | Defects | Nerve conduction | Dorsal root ganglia | Frataxin | Granulocyte colony-stimulating factor | Colony-stimulating factor | Locomotor activity | Medical treatment | Ganglia | Friedreich's ataxia | Gliosis | Ribonucleic acids | Neural stem cells | Leukocytes (granulocytic)
Journal Article
Molecular Diagnosis & Therapy, ISSN 1177-1062, 4/2019, Volume 23, Issue 2, pp. 173 - 186
Inherited monogenic disorders such as beta-hemoglobinopathies (BH) are fitting candidates for treatment via gene therapy by gene transfer or gene editing. The... 
Human Genetics | Pharmacotherapy | Cancer Research | Molecular Medicine | Biomedicine | Laboratory Medicine | LONG-TERM | HUMAN HEMATOPOIETIC STEM | FETAL-HEMOGLOBIN | BONE-MARROW | GENETICS & HEREDITY | PHARMACOLOGY & PHARMACY | SICKLE-CELL-DISEASE | GLOBAL EPIDEMIOLOGY | THALASSEMIA MAJOR | G-CSF | BLOOD PROGENITOR CELLS | LENTIVIRAL VECTORS
Journal Article
International Journal of Radiation Oncology, Biology, Physics, ISSN 0360-3016, 2001, Volume 50, Issue 5, pp. 1161 - 1171
Purpose: To demonstrate the efficacy of radiochemotherapy (RCT) as the first choice of treatment for advanced unresectable head-and-neck cancer. To prove an... 
Head-and-neck cancer | Accelerated radiotherapy | G-CSF | Radiochemotherapy | FRACTIONATION | BOOST | PROGNOSIS | CONVENTIONAL RADIOTHERAPY | CONCOMITANT CHEMORADIOTHERAPY | accelerated radiotherapy | RADIATION-THERAPY | OROPHARYNX | radiochemotherapy | head-and-neck cancer | ONCOLOGY | CONCURRENT CHEMOTHERAPY | SQUAMOUS-CELL CARCINOMA | LOCALLY ADVANCED HEAD | RADIOLOGY, NUCLEAR MEDICINE & MEDICAL IMAGING | Prognosis | Prospective Studies | Follow-Up Studies | Radiotherapy, High-Energy - methods | Humans | Middle Aged | Oropharyngeal Neoplasms - mortality | Male | Hypopharyngeal Neoplasms - mortality | Life Tables | Fluorouracil - administration & dosage | Adult | Female | Chemotherapy, Adjuvant | Oropharyngeal Neoplasms - drug therapy | Head and Neck Neoplasms - drug therapy | Proportional Hazards Models | Carboplatin - administration & dosage | Oropharyngeal Neoplasms - radiotherapy | Treatment Outcome | Combined Modality Therapy | Lymphatic Metastasis | Granulocyte Colony-Stimulating Factor - therapeutic use | Germany - epidemiology | Disease-Free Survival | Head and Neck Neoplasms - radiotherapy | Hypopharyngeal Neoplasms - radiotherapy | Dose Fractionation, Radiation | Antineoplastic Combined Chemotherapy Protocols - therapeutic use | Survival Analysis | Aged | Head and Neck Neoplasms - mortality | Hypopharyngeal Neoplasms - drug therapy | Neoplasm Staging
Journal Article
Leukemia, ISSN 0887-6924, 10/2009, Volume 23, Issue 10, pp. 1904 - 1912
Journal Article