Discover Community Create You

Loading publication...

Noodle always counts identifier-free feature events for service operation. With your permission, Google Analytics can also estimate audience and engagement. Search text, publication identifiers, cookies, IP addresses and browser fingerprints are never included in Noodle's first-party event counters.

Privacy details

Article

Lentiviral Vectors with Cellular Promoters Correct Anemia and Lethal Bone Marrow Failure in a Mouse Model for Diamond-Blackfan Anemia.

Molecular therapy : the journal of the American Society of Gene Therapy - 2 Aug 2017

Debnath Shubhranshu, Jaako Pekka, Siva Kavitha, Rothe Michael, Chen Jun, Dahl Maria, Gaspar H Bobby, Flygare Johan, Schambach Axel, Karlsson Stefan

Abstract excerpt

Diamond-Blackfan anemia is a congenital erythroid hypoplasia and is associated with physical malformations and a predisposition to cancer. Twenty-five percent of patients with Diamond-Blackfan anemia have mutations in a gene encoding ribosomal protein S19 (RPS19). Through overexpression of RPS19 using a lentiviral vector with the spleen focus-forming virus promoter, we demonstrated that the Diamond-Blackfan...

Read the complete abstract on PubMed

Topics

Share this publication in a Topic to start or enrich a Post.

Share to Anemia, Diamond-Blackfan
Share to Bone Marrow Transplantation

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Gene Order

Share to Graft Survival

Share to Hematopoiesis

Share to Lentivirus

Share to Transduction, Genetic

Share to Virus Integration

Source

Open in PubMed Open full text

Identifiers

PMID: 28434866
DOI: 10.1016/j.ymthe.2017.04.002
PMCID: PMC5542636

Genes

RPS19

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Lentiviral Vectors with Cellular Promoters Correct Anemia and Lethal Bone Marrow Failure in a Mouse Model for Diamond-Blackfan Anemia.PMID 28434866Read ↗

Successful gene therapy of Diamond-Blackfan anemia in a mouse model and human CD34+ cord blood hematopoietic stem cells using a clinically applicable lentiviral vector.Semantic proximity Read ↗

Lentivirus-mediated gene therapy corrects ribosomal biogenesis and shows promise for Diamond Blackfan anemia.Semantic proximity Read ↗

Mice with ribosomal protein S19 deficiency develop bone marrow failure and symptoms like patients with Diamond-Blackfan anemia.Semantic proximity Read ↗

Ribosomal and hematopoietic defects in induced pluripotent stem cells derived from Diamond Blackfan anemia patients.Semantic proximity Read ↗

Deficiency of ribosomal protein S19 in CD34+ cells generated by siRNA blocks erythroid development and mimics defects seen in Diamond-Blackfan anemia.Semantic proximity Read ↗

Targeted disruption of the ribosomal protein S19 gene is lethal prior to implantation.Semantic proximity Read ↗

Deficiency of ribosomal protein S19 during early embryogenesis leads to reduction of erythrocytes in a zebrafish model of Diamond-Blackfan anemia.Semantic proximity Read ↗

Enhanced alternative splicing of the FLVCR1 gene in Diamond Blackfan anemia disrupts FLVCR1 expression and function that are critical for erythropoiesis.Semantic proximity Read ↗

Towards a Cure for Diamond-Blackfan Anemia: Views on Gene Therapy.Semantic proximity Read ↗

Generation of iPSC lines and isogenic gene-corrected lines from two individuals with RPS19-mutated Diamond-Blackfan anemia syndrome.Semantic proximity Read ↗

Recent insights into the pathogenesis of Diamond-Blackfan anaemia.Semantic proximity Read ↗

RNA and protein evidence for haplo-insufficiency in Diamond-Blackfan anaemia patients with RPS19 mutations.Semantic proximity Read ↗

⌁Select a neighboring publication to make it the new centre.