Claes Wahlestedt is a Swedish-American biomedical researcher and entrepreneur. He is a professor at the University of Miami. [1]
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A native of Sweden, Wahlestedt obtained his MD and PhD degrees from the University of Lund. He pursued postdoctoral work at Kyoto University and Georgetown University. He grew up in Karlskrona, the son of Åke and Irena Wahlestedt. His father, a lawyer and a decorated former Swedish diplomat, had served in Berlin during the second world war.
Wahlestedt has authored some 300 scientific publications as well as many patents in the fields of biotechnology, genomics and drug discovery. Much of Wahlestedt's career has been devoted to the exploration of non-protein-coding functional elements in the human genome. He is the inventor of the so-called AntagoNAT oligonucleotide technology, which allows for upregulation of genes in genetic and other disorders. [2] [3] Much earlier, in the early 1990s, his team first demonstrated efficacy and utility of antisense oligonucleotides applied directly to the brain (intracerebroventricular or intrathecal). There are today a number of human clinical trials based upon intrathecal or other delivery of oligonucleotides to the brain, including the recent FDA approval of Spinraza. Wahlestedt's team, with collaborators, introduced the now widespread use of locked nucleic acids in oligonucleotides (antisense and later siRNAs). A number of small molecule drugs have also been discovered by or under Wahlestedt's direction. [4] [5]
Currently, at the University of Miami, Wahlestedt is Leonard Miller Professor and Associate Dean for Therapeutic Innovation. Previously, he was a founding faculty member and director of neuroscience research at the Florida campus of The Scripps Research Institute (2005-2011). [6] Before that he was an endowed professor and department chair at the Karolinska Institute in Stockholm (1997-2005), and the founder of the Center for Genomics and Bioinformatics. He has also been a faculty member at Cornell University Medical College and at McGill University. Previously in his career, he spent more than a decade directing large drug discovery and biotechnology teams in the pharmaceutical industry for AstraZeneca (having been the founder of AstraZeneca Research Centre Montreal) and Pharmacia/Pfizer. In recent years he has co-founded several biotechnology companies, including CuRNA Inc., which was acquired by OPKO Health in 2011, [7] Epigenetix Inc. and Jupiter Orphan Therapeutics. [8]
Oligonucleotides are short DNA or RNA molecules, oligomers, that have a wide range of applications in genetic testing, research, and forensics. Commonly made in the laboratory by solid-phase chemical synthesis, these small fragments of nucleic acids can be manufactured as single-stranded molecules with any user-specified sequence, and so are vital for artificial gene synthesis, polymerase chain reaction (PCR), DNA sequencing, molecular cloning and as molecular probes. In nature, oligonucleotides are usually found as small RNA molecules that function in the regulation of gene expression, or are degradation intermediates derived from the breakdown of larger nucleic acid molecules.
Gene silencing is the regulation of gene expression in a cell to prevent the expression of a certain gene. Gene silencing can occur during either transcription or translation and is often used in research. In particular, methods used to silence genes are being increasingly used to produce therapeutics to combat cancer and other diseases, such as infectious diseases and neurodegenerative disorders.
AstraZeneca plc (AZ) is a British-Swedish multinational pharmaceutical and biotechnology company with its headquarters at the Cambridge Biomedical Campus in Cambridge, England. It has a portfolio of products for major diseases in areas including oncology, cardiovascular, gastrointestinal, infection, neuroscience, respiratory, and inflammation. It has been involved in developing the Oxford–AstraZeneca COVID-19 vaccine.
Gene knockdown is an experimental technique by which the expression of one or more of an organism's genes is reduced. The reduction can occur either through genetic modification or by treatment with a reagent such as a short DNA or RNA oligonucleotide that has a sequence complementary to either gene or an mRNA transcript.
Scripps Research, previously known as The Scripps Research Institute (TSRI), is a nonprofit American medical research facility that focuses on research and education in the biomedical sciences. Headquartered in San Diego, California, the institute has over 170 laboratories employing 2,100 scientists, technicians, graduate students, and administrative and other staff.
Antisense therapy is a form of treatment that uses antisense oligonucleotides (ASOs) to target messenger RNA (mRNA). ASOs are capable of altering mRNA expression through a variety of mechanisms, including ribonuclease H mediated decay of the pre-mRNA, direct steric blockage, and exon content modulation through splicing site binding on pre-mRNA. Several ASOs have been approved in the United States, the European Union, and elsewhere.
Antisense RNA (asRNA), also referred to as antisense transcript, natural antisense transcript (NAT) or antisense oligonucleotide, is a single stranded RNA that is complementary to a protein coding messenger RNA (mRNA) with which it hybridizes, and thereby blocks its translation into protein. The asRNAs have been found in both prokaryotes and eukaryotes, and can be classified into short and long non-coding RNAs (ncRNAs). The primary function of asRNA is regulating gene expression. asRNAs may also be produced synthetically and have found wide spread use as research tools for gene knockdown. They may also have therapeutic applications.
Saghir Akhtar is professor of pharmacology in the College of Medicine, Qatar University, and editor in chief of the Journal of Drug Targeting.
In molecular biology and genetics, the sense of a nucleic acid molecule, particularly of a strand of DNA or RNA, refers to the nature of the roles of the strand and its complement in specifying a sequence of amino acids. Depending on the context, sense may have slightly different meanings. For example, the negative-sense strand of DNA is equivalent to the template strand, whereas the positive-sense strand is the non-template strand whose nucleotide sequence is equivalent to the sequence of the mRNA transcript.
Gerald Francis "Jerry" Joyce is president and professor at the Salk Institute for Biological Studies and was previously the director of the Genomics Institute of the Novartis Research Foundation. He is best known for his work on in vitro evolution, for the discovery of the first DNA enzyme (deoxyribozyme), for his work in discovering potential RNA world ribozymes, and more in general for his work on the origin of life.
Rosetta Genomics Ltd. was a molecular diagnostics company with offices in Israel and the United States that uses micro-ribonucleic acid (microRNA) biomarkers to develop diagnostic tests designed to differentiate between various types of cancer. The company expects the first three tests based on its technology to be submitted for regulatory approval in 2008. The diagnostic tests will differentiate between squamous and non-squamous non-small cell lung cancer (NSCLC); differentiate between adenocarcinoma and peritoneal mesothelioma; and seek to identify the origin of tumors in patients representing cancer of unknown primary (CUP). Using a single microRNA, the highly sensitive, highly specific test for squamous and non-squamous lung cancer has passed the prevalidation phase and has been submitted for approval to the New York State Department of Health Clinical Laboratory Evaluation Program in April 2008.
Xenbase is a Model Organism Database (MOD), providing informatics resources, as well as genomic and biological data on Xenopus frogs. Xenbase has been available since 1999, and covers both X. laevis and X. tropicalis Xenopus varieties. As of 2013 all of its services are running on virtual machines in a private cloud environment, making it one of the first MODs to do so. Other than hosting genomics data and tools, Xenbase supports the Xenopus research community though profiles for researchers and laboratories, and job and events postings.
Dietrich A. Stephan is an American human geneticist and entrepreneur who works in personalized medicine. Stephan is currently CEO of NeuBase Therapeutics and a General Partner in Cyto Ventures. Before NeuBase, Stephan was CEO of LifeX and Chairman and Professor of Human Genetics at the University of Pittsburgh. Prior, he was founding Chairman of the Neurogenomics Division at the Translational Genomics Research Institute. Stephan has founded or co-founded 14 biotechnology companies and advised many others. Stephan was co-founder of Navigenics, a personal genetics company.
John B. Hogenesch is an American chronobiologist and Professor of Pediatrics at the Cincinnati Children's Hospital Medical Center. The primary focus of his work has been studying the network of mammalian clock genes from the genomic and computational perspective to further the understanding of circadian behavior. He is currently the Deputy Director of the Center for Chronobiology, an Ohio Eminent Scholar, and Professor of Pediatrics in the Divisions of Perinatal Biology and Immunobiology at the Cincinnati Children's Hospital Medical Center.
Persomics is a private life science company specializing in genetic research, with locations in Boston, MA, Cupertino, CA, and Gothenburg, Sweden. The company’s aim is to simplify and expedite the process of discovering the basis of disease, and has established a particular phenotypic screening kit that it uses for this purpose. These kits facilitate the interrogation of thousands of genes in parallel, and therefore pertain to human genome, pharmaceutical and disease model research.
Gregory James Hannon is a professor of molecular cancer biology and director of the Cancer Research UK Cambridge Institute at the University of Cambridge. He is a Fellow of Trinity College, Cambridge while also serving as a director of cancer genomics at the New York Genome Center and an adjunct professor at Cold Spring Harbor Laboratory.
Gapmers are short DNA antisense oligonucleotide structures with RNA-like segments on both sides of the sequence. These linear pieces of genetic information are designed to hybridize to a target piece of RNA and silence the gene through the induction of RNase H cleavage. Binding of the gapmer to the target has a higher affinity due to the modified RNA flanking regions, as well as resistance to degradation by nucleases. Gapmers are currently being developed as therapeutics for a variety of cancers, viruses, and other chronic genetic disorders.
Ruth Eleanor March is a British genomic scientist who is senior vice president of precision medicine at AstraZeneca. She specialises in precision medicine and oncology. During the COVID-19 pandemic, March developed a diagnostic test for COVID-19.
A majority of the human genome is made up of non-protein coding DNA. It infers that such sequences are not commonly employed to encode for a protein. However, even though these regions do not code for protein, they have other functions and carry necessary regulatory information.They can be classified based on the size of the ncRNA. Small noncoding RNA is usually categorized as being under 200 bp in length, whereas long noncoding RNA is greater than 200bp. In addition, they can be categorized by their function within the cell; Infrastructural and Regulatory ncRNAs. Infrastructural ncRNAs seem to have a housekeeping role in translation and splicing and include species such as rRNA, tRNA, snRNA.Regulatory ncRNAs are involved in the modification of other RNAs.
Stephen Donald Wilton, also known as Steve Wilton, is an Australian molecular biologist and academic, serving as the Foundation Professor of Molecular Therapy at Murdoch University and adjunct professor at the University of Western Australia (UWA). He also fulfills dual roles as a Director at the Perron Institute for Neurological and Translational Science and deputy director at Murdoch's Centre for Molecular Medicine and Innovative Therapeutics (CMMIT).
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