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Research Technology / Nucleic Acid / Antisense Technology

Progress and Promise: The Current Status of Spinal Muscular Atrophy Therapeutics

Abstract: Spinal muscular atrophy (SMA) is an inherited neuromuscular disorder that causes degeneration of α-motor neurons. Frequently, muscle weakness is very severe causing affected infants to die before reaching two years of age, but mild forms of the disease can be characterized by relatively static muscle weakness for many years. SMA is caused by recessive mutations of the SMN1 gene, but all patients retain at least one copy of SMN2, a similar gene capable of producing low levels of full-length SMN protein. No treatments currently exist for SMA patients, but the identification of therapeutic targets and the development of suitable animal models for preclinical testing have resulted in increased drug development efforts in the past ten years. Here, we review the current status of many of these programs, including those designed to activate SMN2 gene expression, modulate splicing of SMN2 preRNAs, stabilize SMN protein, replace SMN1, provide neuroprotective support, and transplant neural cells. ... Read more

The Therapeutic Potential of microRNA Modulation

Abstract: microRNAs are endogenous small non-coding RNAs that regulate gene expression by interfering with translation or stability of target transcripts. The importance and varied functions of microRNAs are illustrated by the diverse phenotypes, including disease, that arise when microRNAs are mutated or improperly expressed. The association of microRNA dysfunction with disease phenotypes has given rise to the idea that selective modulation of microRNAs could alter the course of disease. With the recent demonstration that inhibition of miR-122 reduces viral load in HCV-infected chimpanzees, microRNA modulators are no longer merely theoretical, but have become strong candidate therapeutics. Here we review the evidence for microRNA dysfunction in human disease, as well as recent examples of microRNA modulation that provided therapeutic benefit. ... Read more

Magnetic Nanoparticles: Prospects in Cancer Imaging and Therapy

Abstract: Nanotechnology based on the use of submicronic particles of inorganic and/or organic origin has the potential to revolutionalize the clinical management of cancer; the possibility of real time monitoring of disease progression and effects of therapy is now real. Especially, iron oxide super paramagnetic nanoparticles have shown clinical utility in cancer imaging and drug delivery and some formulations are now FDA-approved for use in the clinic. The prospects of magnetic nanoparticles in cancer imaging and treatment are reviewed. ... Read more

Can We Faithfully Recall What We Remembered?

Abstract: Fresh memories undergo a consolidation process to become permanent. The consolidation process, in which original patterns of synaptic connections (neuron networks) are retrieved and new patterns are formed, is subject to disruption by distracting stimuli, pharmaceutical agents, and toxins, among others. The consolidated memory may, at times, be different from the original one. ... Read more

An Experimental Approach for Systematic Identification of Antisense Transcripts

Natural antisense transcripts are endogenous transcripts that contain sequences that are complementary to other transcripts. Such complementary transcripts may be transcribed from opposing DNA strands at the same genomic locus (cis), or from different loci (trans), for example, pseudogenes. Notably, both types of antisense RNAs are genome-encoded and transcribed by DNA-directed RNA polymerases. A third putative source of antisense RNAs is transcription of the sense mRNA by an RNA-dependent RNA polymerase (Volloch et al., 1996).

Although only a few examples have been studied in detail (reviewed in Kumar and Carmichael, 1998; Lavorgna et al., 2004), a significant number of naturally occurring ... Read more

Antisense Therapy Corrects Nonsense Mutation by Exon Skipping

Antisense technology is based on the principle that antisense oligonucleotides (AO) can specifically alter gene expression and the pattern of splicing at the RNA level. The first application of such technology 25 years ago was for knockdown of targeted viral gene expression (Zamecnik et al., 1978), which was achieved by targeting mRNA with AO to block the viral gene translation or induce its degradation. Such strategies have now been developed as powerful tools for the functional analysis of targeted genes in the laboratory and are suggested to be of great therapeutic potential against diseases from viral infections to cancers. More ... Read more

Antisense therapeutics show promise in cancer treatment

Antisense technology is based on a simple and beautiful concept: a short stretch of DNA, whose sequence is complementary to a specific mRNA of interest, binds to the target and induces its degradation or blocks its expression. Since the first application of antisense DNA on Rous sarcoma virus expression in 1978, numerous DNA oligos have undergone clinical trials. In over two decades of developing this technology, scientists have accumulated a lot of valuable information.

In order to avoid degradation, antisense DNA oligos have to be modified to increase ... Read more

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