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In recent webinars by Genuity Science, formerly known as WuXi NextCODE, experts from the biotech and pharma industries spoke about leveraging the power of single cell RNA sequencing platforms and solutions in conjunction with machine learning technologies such as AI in cell biology and disease research. The Power of Single Cell Technology.
Molecular diagnostic tests refer to tests intended to detect specific sequences in human genomic samples, such as DNA or RNA, in order to diagnose a particular disease. According to the World Health Organization, there are over 40,000 in vitro diagnostics products available in the market. What are Molecular Diagnostic Tests?
The current study extends on that finding and provides crucial mechanistic and clinical insights into the beneficial effects of verapamil in Type 1 diabetes, using proteomics analysis and RNA sequencing. But the preservation of some beta cell function is promising. ” Source link: [link].
The primary endpoint of this trial is to evaluate patients for complete histological clearance of the tumor cells within the treated BCC lesion with secondary endpoints, evaluating subjects for investigational product treatment related adverse events, as well as serious adverse events, and cutaneous skin reactions. Chief Medical Officer.
Of all the Angelman syndrome pipeline therapies , the most touted treatment was Gaboxadol ( OV101 or THIP ), a product of Ovid Therapeutics. For instance, GTX-102 , an antisense therapy silencing the geneexpression by binding to the RNA molecule behind the regulation of its expression, has also secured USFDA Fast Track Designation.
Gene Therapy Definition. Gene therapy is a new therapeutic approach in which genes are used to treat or prevent diseases. It is a comprehensive term which encompasses a large variety of therapy products including viral and bacterial vectors, plasmid DNA, human gene editing technology, and patient-specific cellular gene therapy.
RNA therapeutics are a novel class of biopharmaceuticals that harness the power of RNA molecules for the treatment and prevention of a wide range of disorders, including oncological, and genetic disorders as well as infectious diseases. Non-coding RNAs include antisense oligonucleotides (ASOs) and RNA aptamers.
Innovation S-curve for the pharmaceutical industry Transcription factors for genetically modified cells is a key innovation area in the pharmaceutical industry Transcription factors are proteins which control geneexpression by controlling the rate of transcription of genetic information from DNA to RNA.
The 2024 Nobel Prize in Physiology or Medicine has been awarded to American scientists Victor Ambros and Gary Ruvkun for their groundbreaking discovery of microRNA (or miRNA) and its role in post-transcriptional gene regulation. Their ability to modulate geneexpression at the RNA level provides a novel therapeutic approach for conditions.
Mechanism of Gene Switch During the transcription process, the promoter region, which is located near the upstream end of each gene, binds to transcription factor, which is a specific type of protein. An additional level of genetic control is provided by gene switches that are located upstream of the promoter region.
A female’s second X chromosome is typically silenced (so that females don’t get an overdose of X chromosome genes) through X-linked inactivation, a process where the chromosome is coated with an outer layer of non-coding RNA. One of these doubly active genes is KDM6A. KDM6A Variant.
Galidesivir is a broad-spectrum antiviral, an adenosine nucleoside analog that blocks viral RNA polymerase. STP705 is a small interfering RNA (siRNA) therapy that leverages a dual-targeted inhibitory property and polypeptide nanoparticle (PNP)-enhanced delivery to knock down both TGF-beta1 and COX-2 geneexpression.
From rare disease drug approvals to treatments involving immunotherapies and gene therapies and awarding of a Nobel Prize to the inventors of the gene-editing tool CRISPR, 2020 was a year of great activity and productivity despite the backdrop of the pandemic. Single-Cell RNA Sequencing.
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