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Gene Therapy sentence examples within recombinant adeno associated



Immune responses to retinal gene therapy using adeno-associated viral vectors – Implications for treatment success and safety



Administration of an adeno-associated viral vector expressing interferon-β in patients with inflammatory hand arthritis, results of a phase I/II study.


Gene Therapy sentence examples within hematopoietic stem cell



Engineered stem cells combine stem cell and gene therapy approaches to move intestine therapy from bench to bed



A systematic review of quality of life in sickle cell disease and thalassemia after stem cell transplant or gene therapy.


Gene Therapy sentence examples within spinal muscular atrophy



Newborn screening for spinal muscular atrophy with disease-modifying therapies: a cost-effectiveness analysis



Early Cost-Effectiveness of Onasemnogene Abeparvovec-xioi (Zolgensma) and Nusinersen (Spinraza) Treatment for Spinal Muscular Atrophy I in The Netherlands With Relapse Scenarios.




Gene Therapy sentence examples within sickle cell disease



Sustained fetal hemoglobin induction in vivo is achieved by BCL11A interference and coexpressed truncated erythropoietin receptor



Introduction to a Review Series on gene therapy and gene editing for SCD and hemophilia.


Gene Therapy sentence examples within stem cell therapy



Review on novel targets and therapies for Keloids.



Regenerative technology to restore and preserve erectile function in men following prostate cancer treatment: evidence for penile rehabilitation in the context of prostate cancer survivorship


Gene Therapy sentence examples within adeno associated viral



Preclinical biodistribution, tropism, and efficacy of oligotropic AAV/Olig001 in a mouse model of congenital white matter disease



Proteomic Landscape of Adeno-Associated Virus (AAV)-Producing HEK293 Cells


Gene Therapy sentence examples within central nervous system



Canavan Disease as a Model for Gene Therapy-Mediated Myelin Repair



Gene therapy strategies for rare monogenic disorders with nuclear or mitochondrial gene mutations.


Gene Therapy sentence examples within inherited retinal disease



Gene Therapy for Rdh12-Associated Retinal Diseases Helps to Delay Retinal Degeneration and Vision Loss



Frequency and phenotypic characteristics of RPE65 mutations in the Chinese population


Gene Therapy sentence examples within substrate reduction therapy



Contemporary therapeutics and new drug developments for treatment of Fabry disease: a narrative review.



GM1 Gangliosidosis: Mechanisms and Management


Gene Therapy sentence examples within genome editing tool



Production of Baculovirus and Stem Cells for Baculovirus-Mediated Gene Transfer into Human Mesenchymal Stem Cells.



Predicting base editing outcomes with an attention-based deep learning algorithm trained on high-throughput target library screens


Gene Therapy sentence examples within associated virus serotype



Complete Correction of Brain and Spinal Cord Pathology in Metachromatic Leukodystrophy Mice



Adeno-associated virus serotype 9 antibodies in patients screened for treatment with onasemnogene abeparvovec


Gene Therapy sentence examples within enzyme replacement therapy



Enzyme Replacement Therapy for Succinic Semialdehyde Dehydrogenase Deficiency: Relevance in γ-Aminobutyric Acid Plasticity.



Induction of Tolerance to Therapeutic Proteins With Antigen-Processing Independent T Cell Epitopes: Controlling Immune Responses to Biologics


Gene Therapy sentence examples within bone marrow transplantation



Management of adrenoleukodystrophy: From pre-clinical studies to the development of new therapies.



Investigational Treatments for Epidermolysis Bullosa


Gene Therapy sentence examples within clustered regularly interspaced



Understanding the Genetic and Molecular Basis of Familial Hypertrophic Cardiomyopathy and the Current Trends in Gene Therapy for Its Management



Genome editing using CRISPR/Cas9 to treat hereditary hematological disorders.


Gene Therapy sentence examples within induced pluripotent stem



Corneal endothelial dysfunction: Evolving understanding and treatment options



Gene therapy restores dopamine transporter expression and ameliorates pathology in iPSC and mouse models of infantile parkinsonism


Gene Therapy sentence examples within simplex virus thymidine



The clinical course and management of cervical cancer with splenic metastasis: Case report and review of the literature



Iron Oxide Nanoparticles Promote Cx43-Overexpression of Mesenchymal Stem Cells for Efficient Suicide Gene Therapy during Glioma Treatment


Gene Therapy sentence examples within chimeric antigen receptor



On the Relationship of Viral Particles and Extracellular Vesicles: Implications for Viral Vector Technology



4SCAR2.0: a multi-CAR-T therapy regimen for the treatment of relapsed/refractory B cell lymphomas


Gene Therapy sentence examples within vascular endothelial growth



Long-term safety and efficacy of intramyocardial adenovirus-mediated VEGF-DΔNΔC gene therapy eight-year follow-up of phase I KAT301 study.



Effects of silencing epididymal vascular endothelial growth factor (VEGF) expression on hyaluronidase (HYD) activity in arsenic poisoning rats through downregulating VEGF receptor 2 (VEGFR2).


Gene Therapy sentence examples within gene delivery vector



Polyethylenimine-Modified Bombyx mori Silk Fibroin as a Delivery Carrier of the ING4-IL-24 Coexpression Plasmid



Optimised protocol for accurate titration of AAV vectors.


Gene Therapy sentence examples within autologous hematopoietic stem



Recent Advancements in the Diagnosis and Treatment of Leukodystrophies.



Transfusion and Cellular Therapy in Pediatric Sickle Cell Disease.


Gene Therapy sentence examples within stem cell transplantation



Retiring as a Physician during the 2020 Coronavirus Pandemic



The pathophysiology of immunoporosis: innovative therapeutic targets.


Gene Therapy sentence examples within therapy cell therapy



Review of the Latest Methods of Epidermolysis Bullosa and Other Chronic Wounds Treatment Including BIOOPA Dressing



A review of possible therapies for multiple sclerosis.


Gene Therapy sentence examples within rapidly developing field



Quantitative single-cell transcriptome-based ranking of engineered AAVs in human retinal explants



A native mass spectrometry-based assay for rapid assessment of the empty/full capsid ratio in AAV gene therapy products


Gene Therapy sentence examples within targeted drug delivery



Emerging Applications of Nanotechnology in Healthcare Systems: Grand Challenges and Perspectives



Virus-like particles: preparation, immunogenicity and their roles as nanovaccines and drug nanocarriers


Gene Therapy sentence examples within gene editing tool



Functionalized PDA/DEX-PEI@HA nanoparticles combined with sleeping-beauty transposons for multistage targeted delivery of CRISPR/Cas9 gene.



CRISPR/ Cas9 Off-Targets: Computational Analysis of Causes, Prediction, Detection and Overcoming Strategies


Gene Therapy sentence examples within shown great promise



Knowledge From London and Berlin: Finding Threads to a Functional HIV Cure



Viral-Vector-Delivered Anti-Angiogenic Therapies to the Eye


Gene Therapy sentence examples within late phase clinical



Update on Gene Therapy Clinical Trials for Choroideremia and Potential Experimental Therapies



Treatment of haemophilia: From replacement to gene therapy.


Gene Therapy sentence examples within haematopoietic stem cell



ABCD1 gene mutation in an Italian family with X-linkedadrenoleukodystrophy: case series



Increased incidence of autoimmune cytopenias after allogeneic haematopoietic stem cell transplantation using a matched unrelated donor in children with β‐thalassaemia


Gene Therapy sentence examples within magnetic resonance imaging



A review: Development of magnetic nano vectors for biomedical applications



Tumor Diagnosis and Therapy Mediated by Metal Phosphorus-Based Nanomaterials.


Gene Therapy sentence examples within growth factor therapy



Small molecule-based treatment approaches for intervertebral disc degeneration: Current options and future directions



Plant-Based Natural Products For Wound Healing: A Critical Review


Gene Therapy sentence examples within somatic cell therapy



Evaluation of Advanced Therapy Medicinal Products by the National Institute for Health and Care Excellence (NICE): An Updated Review



Stem cell and tissue engineering approaches in pressure ulcer treatment.


Gene Therapy sentence examples within treat inherited retinal



Optimized adeno-associated virus vectors for efficient transduction of human retinal organoids.



Creating an Ocular Biofactory: Surgical Approaches in Gene Therapy for Acquired Retinal Diseases.


Gene Therapy sentence examples within triple negative breast



Identification of CCNB2 expression in triple-negative breast cancer based on bioinformatics results



Smart Manganese Dioxide-Based Lanthanide Nanoprobes for Triple-Negative Breast Cancer Precise Gene Synergistic Chemodynamic Therapy.


Gene Therapy sentence examples within viral vector delivery



Extensive rod and cone photoreceptor-cell degeneration in rat models of giant axonal neuropathy: implications for gene therapy of human disease



Lentivirus- or AAV-mediated gene therapy interventions in ischemic stroke: A systematic review of preclinical in vivo studies.


Gene Therapy sentence examples within photodynamic therapy photothermal



Tumoral delivery of nanotherapeutics



Recent advances in assembled AIEgens for image-guided anticancer therapy


Gene Therapy sentence examples within allogeneic hematopoietic stem



A Promising Option for ADA‐SCID Patients



Correction of murine sickle cell disease by allogeneic haematopoietic cell transplantation with anti-3rd party veto cells


Gene Therapy sentence examples within mesenchymal stromal cell



Medical Journals Should Be a Forum for Disruptive Research.



Mesenchymal stromal cell variables influencing clinical potency: the impact of viability, fitness, route of administration and host predisposition.


Gene Therapy sentence examples within chemotherapy photothermal therapy



Multiple-therapy strategies via polysaccharides-based nano-systems in fighting cancer.



Injectable thermosensitive hydrogel-based drug delivery system for local cancer therapy.


Gene Therapy sentence examples within blood brain barrier



Substance P containing peptide gene delivery vectors for specifically transfecting glioma cells mediated by a neurokinin-1 receptor.



Short regulatory DNA sequences to target brain endothelial cells for gene therapy.


Gene Therapy sentence examples within pre existing immunity



An AAV-based, room-temperature-stable, single-dose COVID-19 vaccine provides durable immunogenicity and protection in non-human primates



In Vitro and In Vivo Evaluation of Human Adenovirus Type 49 as a Vector for Therapeutic Applications


Gene Therapy sentence examples within stem cell replacement



An alternative approach to produce versatile retinal organoids with accelerated ganglion cell development



Human stem cell models of polyglutamine diseases: Sources for disease models and cell therapy


Gene Therapy sentence examples within age related macular



Gene therapy in retinal diseases: A review



Hints for Gentle Submacular Injection in Non-Human Primates Based on Intraoperative OCT Guidance


Gene Therapy sentence examples within long term safety



Enhanced Homology-directed Repair for Highly Efficient Gene Editing in Hematopoietic Stem/Progenitor Cells.



Long-Term Safety Evaluation of Continuous Intraocular Delivery of Aflibercept by the Intravitreal Gene Therapy Candidate ADVM-022 in Nonhuman Primates


Gene Therapy sentence examples within promising therapeutic strategy



Clinical Trials for Gene Therapy in Lysosomal Diseases With CNS Involvement



Base and Prime Editing Technologies for Blood Disorders


Gene Therapy sentence examples within therapy stem cell



Overview of methods for enhancing bone regeneration in distraction osteogenesis: Potential roles of biometals



Design and technical challenge of retinal prosthesis


Gene Therapy sentence examples within stem cell transplant



SARS-CoV-2 infection associated with hepatitis in an infant with X-linked severe combined immunodeficiency



Gene delivery using AAV8 in vivo for disease stabilization in a bimodal gene therapy approach for the treatment of ADA-deficient SCID


Gene Therapy sentence examples within β globin gene



Novel Therapeutic Advances in β-Thalassemia



Wild-type HIV infection after treatment with lentiviral gene therapy for β-thalassemia.


Gene Therapy sentence examples within umbilical cord blood



Combination of epidural electrical stimulation with ex vivo triple gene therapy for spinal cord injury: a proof of principle study



Gene-modified leucoconcentrate for personalized ex vivo gene therapy in a mini pig model of moderate spinal cord injury


Gene Therapy sentence examples within promising therapeutic approach



Synergistic gene delivery by self-assembled nanocomplexes using fusion peptide and calcium phosphate.



Viral Gene Therapy for Glioblastoma Multiforme: A Promising Hope for the Current Dilemma


Gene Therapy sentence examples within survival motor neuron



Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial



Spinal muscular atrophy: From approved therapies to future therapeutic targets for personalized medicine



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