Genetic treatment of a molecular disorder: gene therapy approaches to sickle cell disease.
about
Precipitating factors and targeted therapies in combating the perils of sickle cell disease--- A special nutritional considerationCustomizing the genome as therapy for the β-hemoglobinopathiesA genome editing primer for the hematologistMinireview: Prognostic factors and the response to hydroxurea treatment in sickle cell disease.Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype.Pediatric sickle cell disease: past successes and future challenges.Quantitative assessment of timing, efficiency, specificity and genetic mosaicism of CRISPR/Cas9-mediated gene editing of hemoglobin beta gene in rhesus monkey embryos.CRISPR-Cas9 technology and its application in haematological disorders.Emerging cellular and gene therapies for congenital anemias.Microfluidics for investigating vaso-occlusions in sickle cell disease.Adult haematopoietic stem cell niches.Eliminating HIV-1 Packaging Sequences from Lentiviral Vector Proviruses Enhances Safety and Expedites Gene Transfer for Gene Therapy.Sickle cell disease: an international survey of results of HLA-identical sibling hematopoietic stem cell transplantation.CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells.Impact of different promoters, promoter mutation, and an enhancer on recombinant protein expression in CHO cells.World Sickle Cell Day 2016 : A time for appraisal.Fertility challenges for women with sickle cell disease.Preclinical and clinical advances in transposon-based gene therapy.A genome-editing strategy to treat β-hemoglobinopathies that recapitulates a mutation associated with a benign genetic condition.A cross-sectional clinic-based study exploring whether variants within the glutathione S-transferase, haptoglobin and uridine 5'-diphospho-glucuronosyltransferase 1A1 genes are associated with interindividual phenotypic variation in sickle cell anaeDirect digestion of living cells via a gel-based strategy for mass spectrometric analysis.Therapeutic Peptide Nucleic Acids: Principles, Limitations, and Opportunities.Global Transcriptional Response to CRISPR/Cas9-AAV6-Based Genome Editing in CD34 Hematopoietic Stem and Progenitor Cells
P2860
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P2860
Genetic treatment of a molecular disorder: gene therapy approaches to sickle cell disease.
description
2016 nî lūn-bûn
@nan
2016年の論文
@ja
2016年論文
@yue
2016年論文
@zh-hant
2016年論文
@zh-hk
2016年論文
@zh-mo
2016年論文
@zh-tw
2016年论文
@wuu
2016年论文
@zh
2016年论文
@zh-cn
name
Genetic treatment of a molecul ...... oaches to sickle cell disease.
@ast
Genetic treatment of a molecul ...... oaches to sickle cell disease.
@en
type
label
Genetic treatment of a molecul ...... oaches to sickle cell disease.
@ast
Genetic treatment of a molecul ...... oaches to sickle cell disease.
@en
prefLabel
Genetic treatment of a molecul ...... oaches to sickle cell disease.
@ast
Genetic treatment of a molecul ...... oaches to sickle cell disease.
@en
P2860
P1433
P1476
Genetic treatment of a molecul ...... oaches to sickle cell disease.
@en
P2093
Stuart H Orkin
P2860
P304
P356
10.1182/BLOOD-2015-09-618587
P407
P577
2016-01-12T00:00:00Z