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Editions published in Human Gene Therapy 200
Too many entities requested (4316). Only 200 are displayed.
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Gene Therapy for X-Linked Severe Combined Immunodeficiency: Where Do We Stand?
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FMRP Expression Levels in Mouse Central Nervous System Neurons Determine Behavioral Phenotype.
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Dimeric CRISPR RNA-Guided FokI-dCas9 Nucleases Directed by Truncated gRNAs for Highly Specific Genome Editing
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The Bacterial Origins of the CRISPR Genome-Editing Revolution
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CAR T-Cell Therapy: The Role of Physical Barriers and Immunosuppression in Lymphoma
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Replication-Competent Viruses as Cancer Immunotherapeutics: Emerging Clinical Data
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Oligonucleotide Therapies: The Past and the Present
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Gene therapy of inherited retinal degenerations: prospects and challenges
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Adeno-associated virus at 50: a golden anniversary of discovery, research, and gene therapy success--a personal perspective
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Challenges and Prospects for Alpha-1 Antitrypsin Deficiency Gene Therapy
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Perspectives on best practices for gene therapy programs
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My career path for developing gene therapy for blinding diseases: the importance of mentors, collaborators, and opportunities
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MicroRNA 142-3p attenuates spread of replicating retroviral vector in hematopoietic lineage-derived cells while maintaining an antiviral immune response
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A human ErbB2-specific T-cell receptor confers potent antitumor effector functions in genetically engineered primary cytotoxic lymphocytes
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Inhibitory effect of nuclear factor-κB decoy oligodeoxynucleotide on liver fibrosis through regulation of the epithelial-mesenchymal transition
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Adeno-associated virus serotypes 1, 8, and 9 share conserved mechanisms for anterograde and retrograde axonal transport.
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SERCA2a gene therapy can improve symptomatic heart failure in δ-sarcoglycan-deficient animals.
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From the mouse cage to human therapy: a personal perspective of the emergence of T-bodies/chimeric antigen receptor T cells
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Toward synthetic biology with engineered T cells: a long journey just begun
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Effects of adeno-associated virus serotype and tissue-specific expression on circulating biomarkers of propionic acidemia
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Netrin-1 ameliorates myocardial infarction-induced myocardial injury: mechanisms of action in rats and diabetic mice
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Cell replacement therapies: is it time to reprogram?
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Pretransplant mobilization with granulocyte colony-stimulating factor improves B-cell reconstitution by lentiviral vector gene therapy in SCID-X1 mice
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Circumventing antivector immunity: potential use of nonhuman adenoviral vectors
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Insight concerning the mechanism of therapeutic ultrasound facilitating gene delivery: increasing cell membrane permeability or interfering with intracellular pathways?
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Partial correction of the CNS lysosomal storage defect in a mouse model of juvenile neuronal ceroid lipofuscinosis by neonatal CNS administration of an adeno-associated virus serotype rh.10 vector expressing the human CLN3 gene.
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Treatment of congenital neurotransmitter deficiencies by intracerebral ventricular injection of an adeno-associated virus serotype 9 vector
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Similar therapeutic efficacy between a single administration of gene therapy and multiple administrations of recombinant enzyme in a mouse model of lysosomal storage disease
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Efficient clinical scale gene modification via zinc finger nuclease-targeted disruption of the HIV co-receptor CCR5
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Antisense oligonucleotides for the treatment of spinal muscular atrophy
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Human cone visual pigment deletions spare sufficient photoreceptors to warrant gene therapy
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Gene therapy for rare diseases: summary of a National Institutes of Health workshop, September 13, 2012
Subject - wd:Q15757580