Results 51 to 60 of about 1,703,096 (348)

Silencing disease genes in the laboratory and the clinic

, 2011
Synthetic nucleic acids are commonly used laboratory tools for modulating gene expression and have the potential to be widely used in the clinic. Progress towards nucleic acid drugs, however, has been slow and many challenges remain to be overcome before
Corey, David R., Watts, Jonathan K.
core   +1 more source

Gene Therapy for Cardiovascular Disease [PDF]

, 2003
The last decade has seen substantial advances in the development of gene therapy strategies and vector technology for the treatment of a diverse number of diseases, with a view to translating the successes observed in animal models into the clinic ...
Baker, Andrew H., Denby, Laura, Dishart, Kate L., Work, Lorraine M.   +3 more
core   +4 more sources

Antisense Oligonucleotide-Based Therapy for Neuromuscular Disease

Molecules, 2017
Neuromuscular disorders such as Duchenne Muscular Dystrophy and Spinal Muscular Atrophy are neurodegenerative genetic diseases characterized primarily by muscle weakness and wasting.
Valentina Sardone, Haiyan Zhou, Francesco Muntoni, Alessandra Ferlini, Maria Sofia Falzarano   +4 more
doaj   +1 more source

Efficient Modulation of Exon Skipping via Antisense Circular RNAs

Research, 2023
Splice-switching antisense oligonucleotides (ASOs) and engineered U7 small nuclear ribonucleoprotein (U7 Sm OPT) are the most commonly used methods for exon skipping. However, challenges remain, such as limited organ delivery and repeated dosing for ASOs
Shuaiwei Ren, Mei Huang, Raoxian Bai, Lijiao Chen, Jiao Yang, Junyu Zhang, Wenting Guo, Weizhi Ji, Yongchang Chen   +8 more
doaj   +1 more source

Nucleic Acid Carriers Based on Precise Polymer Conjugates [PDF]

, 2011
Polymer polydispersity, random conjugation of functional groups, and poorly understood structure–activity relationships have constantly hampered progress in the development of nucleic acid carriers.
Troiber, Christina, Wagner, Ernst
core   +1 more source

Antisense Oligonucleotides: Pharmacology and Delivery Strategies

INTERNATIONAL JOURNAL OF APPLIED PHARMACEUTICAL SCIENCES AND RESEARCH, 2020
Antisense oligonucleotide therapy is a dominant drug discovery approach that can explicitly modify protein synthesis through numerous mechanisms. The limitations of antisense oligonucleotide (ASO) therapy in delivery strategies have been overcome in recent years with different ligands carriers, as well as, through nanocarriers.
Samad, Mohammed Abdul, Pandiri, Kavya, Bojanapally, Anjani Prasad   +2 more
openaire   +3 more sources

RNA interference approaches for treatment of HIV-1 infection. [PDF]

, 2015
HIV/AIDS is a chronic and debilitating disease that cannot be cured with current antiretroviral drugs. While combinatorial antiretroviral therapy (cART) can potently suppress HIV-1 replication and delay the onset of AIDS, viral mutagenesis often leads to
Bobbin, Maggie L, Burnett, John C, Rossi, John J   +2 more
core   +1 more source

Survivin as a therapeutic target in Sonic hedgehog-driven medulloblastoma. [PDF]

, 2015
Medulloblastoma (MB) is a highly malignant brain tumor that occurs primarily in children. Although surgery, radiation and high-dose chemotherapy have led to increased survival, many MB patients still die from their disease, and patients who survive ...
Berezov, A, Brun, SN, Conway, EM, Crawford, JR, Esparza, LA, Garcia, G, Grant, GA, Greene, MI, Huang, J-M, Levy, ML, Li, X-N, Markant, SL, Nakano, I, Pavlyukov, MS, Smith, LH, Terry, D, Wang, Q, Wechsler-Reya, RJ   +17 more
core   +2 more sources

Systemic Antisense Therapeutics for Dystrophin and Myostatin Exon Splice Modulation Improve Muscle Pathology of Adult mdx Mice

Molecular Therapy: Nucleic Acids, 2017
Antisense-mediated exon skipping is a promising approach for the treatment of Duchenne muscular dystrophy (DMD), a rare life-threatening genetic disease due to dystrophin deficiency.
Ngoc Lu-Nguyen, Alberto Malerba, Linda Popplewell, Fred Schnell, Gunnar Hanson, George Dickson   +5 more
doaj   +1 more source

LDLR-Gene therapy for familial hypercholesterolaemia: Problems, progress, and perspectives [PDF]

, 2010
Coronary artery diseases (CAD) inflict a heavy economical and social burden on most populations and contribute significantly to their morbidity and mortality rates.
Al-Allaf, FA, Coutelle, C, David, A, Harbottle, R, Themis, M, Waddington, SN   +5 more
core   +3 more sources