Αποτελέσματα Αναζήτησης
3 Φεβ 2021 · We review gene therapy for neuromuscular disorders (spinal muscular atrophy [SMA]; Duchenne muscular dystrophy [DMD]; X-linked myotubular myopathy [XLMTM]; and diseases of the central nervous system, including Alzheimer's disease, Parkinson's disease, Canavan disease, aromatic l-amino acid decarboxylase [AADC] deficiency, and giant axonal ...
8 Νοε 2023 · In this article, we highlight success and ongoing challenges in three areas of high activity in gene therapy: inherited blood cell diseases by targeting hematopoietic stem cells, malignant...
8 Σεπ 2024 · Ex vivo and in vivo somatic gene and cell therapy strategies. This figure illustrates two primary approaches to gene therapy: In vivo and ex vivo. In the in vivo gene therapy (left panel) strategy, a therapeutic gene is first inserted into a suitable vector either viral or non-viral, 1 most commonly employing Adeno-associated viral (AAV ...
31 Μαΐ 2021 · The increasing number of approved nucleic acid therapeutics demonstrates the potential to treat diseases by targeting their genetic blueprints in vivo. Conventional treatments generally...
18 Απρ 2011 · In vivo gene replacement for the treatment of inherited disease is one of the most compelling concepts in modern medicine. Adeno-associated virus (AAV) vectors have been extensively used...
6 Research Department Cell and Gene Therapy, Department of Stem Cell Transplantation ... as prototype disease where ex vivo investigation on primary cells is challenging, but orthotopic PDX models are promising 21,22. In proof of principle ... for library preparation 2,000 cells of each individual sample were sorted and lysed in RLT Plus ...
23 Νοε 2023 · A much broader variety of monogenetic diseases are tackled with in vivo genome editing (table 2). On systemic administration, lipid nanoparticles (LNPs) and viral vectors, including most adeno-associated viral (AAV) serotypes, distribute mainly to the liver.
