Biomaterial Database

A novel nonviral gene delivery system for treating Leber's congenital amaurosis. 2017

Da Sun, and Rebecca M Schur, and Zheng-Rong Lu

Case Center for Biomolecular Engineering, Department of Biomedical Engineering, School of Engineering, Case Western Reserve University, Cleveland, OH 44106, USA.

UI	MeSH Term	Description	Entries
D010957	Plasmids	Extrachromosomal, usually CIRCULAR DNA molecules that are self-replicating and transferable from one organism to another. They are found in a variety of bacterial, archaeal, fungal, algal, and plant species. They are used in GENETIC ENGINEERING as CLONING VECTORS.	Episomes,Episome,Plasmid
D003545	Cysteine	A thiol-containing non-essential amino acid that is oxidized to form CYSTINE.	Cysteine Hydrochloride,Half-Cystine,L-Cysteine,Zinc Cysteinate,Half Cystine,L Cysteine
D004247	DNA	A deoxyribonucleotide polymer that is the primary genetic material of all cells. Eukaryotic and prokaryotic organisms normally contain DNA in a double-stranded state, yet several important biological processes transiently involve single-stranded regions. DNA, which consists of a polysugar-phosphate backbone possessing projections of purines (adenine and guanine) and pyrimidines (thymine and cytosine), forms a double helix that is held together by hydrogen bonds between these purines and pyrimidines (adenine to thymine and guanine to cytosine).	DNA, Double-Stranded,Deoxyribonucleic Acid,ds-DNA,DNA, Double Stranded,Double-Stranded DNA,ds DNA
D005029	Ethylenediamines	Derivatives of ethylenediamine (the structural formula NH2CH2CH2NH2).
D005233	Fatty Alcohols	Usually high-molecular-weight, straight-chain primary alcohols, but can also range from as few as 4 carbons, derived from natural fats and oils, including lauryl, stearyl, oleyl, and linoleyl alcohols. They are used in pharmaceuticals, cosmetics, detergents, plastics, and lube oils and in textile manufacture. (From McGraw-Hill Dictionary of Scientific and Technical Terms, 5th ed)	Fatty Alcohol,Alcohol, Fatty,Alcohols, Fatty
D006801	Humans	Members of the species Homo sapiens.	Homo sapiens,Man (Taxonomy),Human,Man, Modern,Modern Man
D000818	Animals	Unicellular or multicellular, heterotrophic organisms, that have sensation and the power of voluntary movement. Under the older five kingdom paradigm, Animalia was one of the kingdoms. Under the modern three domain model, Animalia represents one of the many groups in the domain EUKARYOTA.	Animal,Metazoa,Animalia
D015316	Genetic Therapy	Techniques and strategies which include the use of coding sequences and other conventional or radical means to transform or modify cells for the purpose of treating or reversing disease conditions.	Gene Therapy,Somatic Gene Therapy,DNA Therapy,Gene Therapy, Somatic,Genetic Therapy, Gametic,Genetic Therapy, Somatic,Therapy, DNA,Therapy, Gene,Therapy, Somatic Gene,Gametic Genetic Therapies,Gametic Genetic Therapy,Genetic Therapies,Genetic Therapies, Gametic,Genetic Therapies, Somatic,Somatic Genetic Therapies,Somatic Genetic Therapy,Therapies, Gametic Genetic,Therapies, Genetic,Therapies, Somatic Genetic,Therapy, Gametic Genetic,Therapy, Genetic,Therapy, Somatic Genetic
D053758	Nanoparticles	Nanometer-sized particles that are nanoscale in three dimensions. They include nanocrystaline materials; NANOCAPSULES; METAL NANOPARTICLES; DENDRIMERS, and QUANTUM DOTS. The uses of nanoparticles include DRUG DELIVERY SYSTEMS and cancer targeting and imaging.	Nanocrystalline Materials,Nanocrystals,Material, Nanocrystalline,Materials, Nanocrystalline,Nanocrystal,Nanocrystalline Material,Nanoparticle
D057130	Leber Congenital Amaurosis	A rare degenerative inherited eye disease that appears at birth or in the first few months of life that results in a loss of vision. Not to be confused with LEBER HEREDITARY OPTIC NEUROPATHY, the disease is thought to be caused by abnormal development of PHOTORECEPTOR CELLS in the RETINA, or by the extremely premature degeneration of retinal cells.	Amaurosis, Leber Congenital,Congenital Amaurosis of Retinal Origin,Congenital Retinal Blindness,Dysgenesis Neuroepithelialis Retinae,Hereditary Epithelial Dysplasia of Retina,Hereditary Retinal Aplasia,Heredoretinopathia Congenitalis,Leber Abiotrophy,Leber Congenital Tapetoretinal Degeneration,Leber's Amaurosis,Abiotrophies, Leber,Abiotrophy, Leber,Amauroses, Leber Congenital,Amauroses, Leber's,Amaurosis, Leber's,Blindness, Congenital Retinal,Blindnesses, Congenital Retinal,Congenital Amauroses, Leber,Congenital Amaurosis, Leber,Congenital Retinal Blindnesses,Leber Abiotrophies,Leber Amaurosis,Leber Congenital Amauroses,Leber's Amauroses,Lebers Amaurosis,Retinal Blindnesses, Congenital