НАСЛЕДСТВЕННЫЕ БОЛЕЗНИ
ТЕРАПИЯ
B
B-CELL LEUCOSIS AND LYMPHOMS
GENE THERAPY
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gene therapy to achieve long-term continuous or controllable expression of biotherapeutics
CELL THERAPY
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Non-viral, specifically targeted CAR-T cells achieve high safety and efficacy in B-NHL
B CELL-BASED GENE THERAPIES
GENE THERAPY
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Vector Strategies to Actualize B Cell-Based Gene Therapies
BASE EDITING DNA
ZINC FINGER DEAMINASES
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Nuclear and mitochondrial DNA editing in human cells with zinc finger deaminases
CHOLESTEROL
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Super-precise gene-editing approach switches off a gene in the liver that regulates ‘bad’ cholesterol.
BASE EDITING mtDNA
TALE-LINKED DEAMINASES
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Engineering TALE-linked deaminases to facilitate precision adenine base editing in mitochondrial DNA
BATTEN DISEASE
CURRENT TREATMENTS
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S
Therapeutic landscape for Batten disease
β-THALASSEMIA
STEM CELL GENE THERAPY
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S
Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent β-thalassemia
TRANSFUSION-DEPENDENT β-THALASSEMIA
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Approach for Transfusion-Dependent β-Thalassemia—CRISPR-Cas9 Gene Editing for β-Thalassemia
TARGETED THERAPY
TARGETED BIOLOGICS
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Targeted Biologics: The New Frontier for Precision Therapy
BILIARY DISEASES
BILIARY FIBROSIS
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S
Current therapies and novel approaches for biliary diseases
3D BIOPRINTING
MODULATION FOR GENETIC EXPRESSION
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S
3D Printed Bioconstructs: Regenerative Modulation for Genetic Expression
BLOOD DISEASES
GENE THERAPY
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S
Gene therapy for blood diseases
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S
Gene therapy and gene editing strategies in inherited blood disorders
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S
Advances in the gene therapy of monogenic blood cell diseases
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S
On gene therapy and gene editing for sickle cell disease and hemophilia
BLOOD SUBSTITUTES
OXYGEN THERAPEUTICS
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S
Supressing the 'radical' nature of Hb
BRAIN DISEASE
GENE THERAPY AND EDITING
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Gene delivery to the brain: silencing and editing
BREAST CANCER
GENE CARRIER
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ROS-Biodegradable Gene Carrier
THERAPY
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Liposomes and Their Advancements as a Form of Gene Therapy Treatment for Breast Cancer
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Navigating the therapeutic landscape for breast cancer: targeting breast cancer stem cells