• Next generation modified antisense oligonucleotides (ASOs) are commercially approved new therapeutic modalities, yet poor productive uptake and endosomal entrapment in tumour cells limit their broad application. (bvsalud.org)
  • Cell-penetrating peptides (CPPs) was conjugated on HepG2 cells-derived exosomes surface which can not only enhance the penetrating capacity of exosomes but also assist exosomes in loading antisense oligonucleotides (ASOs). (bvsalud.org)
  • Nucleic acids, such as antisense oligonucleotides (AONs) and small interfering RNAs (siRNAs) have enormous potential as therapeutics for life-threatening diseases because in principle, any problematic gene can be targeted with high efficacy and sequence-specificity. (grstiftung.ch)
  • A wide range of possible antisense LNA oligonucleotides were described in the applicants' earlier international patent application publication No. WO 2004/069991 A2. (justia.com)
  • Many oligo backbone modifications create unique binding capabilities and bolster sequence discrimination, making them useful for a variety of diagnostic and therapeutic applications such as SNP genotyping, antisense oligonucleotides, siRNA, and cell delivery. (biosearchtech.com)
  • By involving academics and industry , we aim to assess the state of the art of the delivery of Antisense Oligonucleotides (ASOs) (read more in the sections below) and identify gaps in the knowledge to prioritize and align research efforts. (antisenserna.eu)
  • Antisense oligonucleotides (ASOs) are a new class of drugs that, through precise targeting in RNA strands , could correct genetic defects for rare inherited diseases , modulate autoimmune or neurodegenerative diseases or target tumours or viruses . (antisenserna.eu)
  • Inactivating Gene Expression with Antisense Modified Oligonucleotides. (yale.edu)
  • Mesyl phosphoramidate backbone modified antisense oligonucleotides targeting miR-21 with enhanced in vivo therapeutic potency. (yale.edu)
  • They include coding mRNAs and non-coding (nc) RNAs among them antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), micro-RNAs (miRNAs), small activating RNAs (saRNAs), RNA aptamers and RNA guides. (frontiersin.org)
  • There are currently several products containing RNA on the market, and many are under development, among them mRNA, antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), small activating RNAs (saRNAs), micro-RNAs (mi-RNAs), RNA aptamers and RNA guides. (frontiersin.org)
  • They readily bind to complementary oligonucleotides or to nucleic acids, forming a duplex. (biologyonline.com)
  • Aptamers (from the Latin aptus ("- fit") and Greek meros ("-part") that are oligonucleotides (since other aptamers can be peptide molecules) are created usually from a large random sequence pool and used mainly for research and clinical applications (e.g. as macromolecular drugs). (biologyonline.com)
  • Antisense oligonucleotides are DNA or RNA strands that are used in antisense therapy. (biologyonline.com)
  • Delivery of therapeutic oligonucleotides with cell penetrating peptides. (ox.ac.uk)
  • This review focuses on CPP-delivery of several classes of oligonucleotides (ONs), namely antisense oligonucleotides, splice switching oligonucleotides (SSOs) and siRNAs. (ox.ac.uk)
  • Comparisons of PNA effects on sense and antisense transcription of the AR, TBP, and c-myc genes confirm that progression of the RNA polymerase complex beyond the site of PNA⅐DNA hybridization is impaired in both directions. (archive.org)
  • Many scientists are still confused by the terms sense and antisense when referring to DNA because the terminology has changed over the years. (genomicglossaries.com)
  • In addition to the well-established sense-antisense complementarity abundantly present in the nucleic acid world and serving as a basic principle of the specific double-helical structure of DNA, production of mRNA, and genetic code-based biosynthesis of proteins, sense-antisense complementarity is also present in proteins, where sense and antisense peptides were shown to interact with each other with increased probability. (usf.edu)
  • In proteins, the complementarity between sense and antisense peptides depends on a specific hydropathic pattern, where codons for hydrophilic and hydrophobic amino acids in a sense peptide are complemented by the codons for hydrophobic and hydrophilic amino acids in its antisense counterpart. (usf.edu)
  • We are showing here that in addition to this pattern of the complementary hydrophobicity, sense and antisense peptides are characterized by the complementary order-disorder patterns and show complementarity in sequence distribution of their disorder-based interaction sites. (usf.edu)
  • In particular, the FANTOM (Functional Annotation of the Mammalian Genome) consortium pioneered the discovery of the noncoding RNA world by providing, through Cap Analysis of Gene Expression (CAGE-Seq), the first evidence that large portions of our genome are transcribed, producing a multitude of sense and antisense transcripts 12 . (nature.com)
  • Here we compare intracellular traffic of anti KRAS antisense oligonucleotide (AZD4785) in tumour cell lines PC9 and LK2, with good and poor productive uptake, respectively. (bvsalud.org)
  • Peptide nucleic acid (PNA) is an artificial oligonucleotide mimetic with a peptidic backbone in lieu of a phosphoribosyl backbone. (creative-peptides.com)
  • Generate uncharged and nuclease-resistant oligonucleotide linkages- particularly useful for targeted cellular delivery of antisense therapeutic agents. (biosearchtech.com)
  • Antisense oligonucleotide gapmers containing phosphoryl guanidine groups reverse MDR1-mediated multiple drug resistance of tumor cells. (yale.edu)
  • Subsequent antisense oligonucleotide-knockdown transcriptomic profiling of two LEC- and two BEC-specific lncRNAs identifies LETR1 as a critical gatekeeper of the global LEC transcriptome. (nature.com)
  • In molecular genetics, they refer to a short sequence synthesised to match a regionwhere a mutation is known to occur, and then used as a probe (oligonucleotide probes). (biologyonline.com)
  • Apart from the long chain of nucleic acids, nucleotides also occur in cyclic forms. (biologyonline.com)
  • Modified RNA molecules, antisense nucleotides, and iRNAs are becoming more and more popular with their ability to control protein expression. (biopharmatrend.com)
  • We investigate the reaction conditions of the sortase-mediated ligation and demonstrate a useful application by the synthesis of a peptide nucleic acid-cell-penetrating peptide chimera, the reaction equilibrium of which can be shifted in favor of the product by dialyzing out the low molecular weight byproduct. (nih.gov)
  • The antisense strand is the template for mRNA synthesis. (genomicglossaries.com)
  • 9. Synthesis of protected pseudopeptides from dicarboxylic amino acids by Mitsunobu condensation / N. P. Boyarskaya, D. I. Prokhorov, Yu. (finechem-mirea.ru)
  • Monomer for Me-phosphonate nucleic acid synthesis. (biosearchtech.com)
  • Synthesis resembles peptide synthesis. (biosearchtech.com)
  • Peptide nucleic acid (PNA) amphiphiles: synthesis, self-assembly, and duplex stability. (umassmed.edu)
  • Both linear and cyclic peptides have been explored as trafficking moiety due to ease of synthesis, structural simplicity, and low probability of undesirable immunogenicity. (biosyn.com)
  • Oligomers are produced synthetically through solid-phase chemical synthesis for use in PCR (polymerase chain reaction, where the oligosaccharide used is referred to as the primer ), DNA sequencing, DNA microarrays, Southern blots, ASO analysis, FISH (florescent in situ hybridization), molecular probing, and library construction. (biologyonline.com)
  • The synthesis and applications of the peptides are gaining increasing popularity as a result of the developments in biotechnology and bioengineering areas and for a number of research purposes including cancer diagnosis and treatment, antibiotic drug development, epitope mapping, production of antibodies, and vaccine design. (intechopen.com)
  • The aim of this chapter is to review some applications of synthetic peptides providing a brief knowledge about peptide synthesis. (intechopen.com)
  • In the first part, information about the peptide synthesis was given in a very simple and readable format under the title of solid-phase peptide synthesis including a brief history, solid supports, linkers, protecting groups, and analysis method sections. (intechopen.com)
  • The synthesis of the peptides may provide sufficient material to enable further studies and to determine the structure-activity relationships or may provide discovery of new analogues with improved properties [ 1 , 2 , 3 , 4 , 5 , 6 , 7 ]. (intechopen.com)
  • Backbone modifications for enhanced cellular uptake: For enhanced cellular delivery, PNA has been conjugated with cell penetrating peptide (CPP), cationic peptides, and basic amino acid stretches such as Lysine and Arginine. (creative-peptides.com)
  • NA sequences downstream of the site of PNA⅐DNA hybridization, but not upstream of that site. (archive.org)
  • Each unit is linked to an appropriate purine or pyrimidine base to create the sequence required for hybridization to the targeted nucleic acid (1-3). (archive.org)
  • Backbone modification and Hybridization properties: Hence the enhancement of binding affinity and sequence specificity of PNA for DNA and RNA complement via chemical modification of PNA backbone is highly desirable and will have significant contribution for potential application in therapeutics and diagnostics. (creative-peptides.com)
  • in vitro, they are formed by nucleic acid hybridization. (umassmed.edu)
  • Global Nucleic Acid Therapeutics CDMO Market to Reach $14.19 Billion by 2033. (gii.tw)
  • The global nucleic acid therapeutics CDMO market was valued at $3.88 billion in 2022 and is expected to reach $14.19 billion by 2033, growing at a CAGR of 12.55% during the forecast period 2023-2033. (gii.tw)
  • The key factors driving the growth of the global nucleic acid therapeutics CDMO market include the growing demand for nucleic acid therapeutics applications to treat chronic and genetic diseases, manufacturing advancement for producing nucleic acid by CDMO (a contract development and manufacturing organization), increasing FDA (Food and Drug Administration) approvals of nucleic acid therapeutics, and increasing investment for the expansion of CDMO manufacturing units. (gii.tw)
  • According to BIS Research , the nucleic acid therapeutics CDMO market refers to the products and services provided by CDMO companies for the manufacturing of nucleic acid therapeutics. (gii.tw)
  • Fusion proteins, peptides, and peptibodies (proteins fused to antibodies) form a separate group of therapeutics. (biopharmatrend.com)
  • Moreover, as the word 'coding' refers to the relationship between nucleic acids and proteins, rather than the mere transcription of DNA into RNA, it is logical to call the strand with the mRNA sequence the coding strand, as in the first example. (genomicglossaries.com)
  • Peptides derived from sequence of cell surface proteins, such as intercellular adhesion molecule-1 (ICAM-1), LHRH, Bombesin, and LFA-1, have shown potent binding affinity to the target cell surface receptors. (biosyn.com)
  • Arg-Gly-Asp (RGD) peptides,2 poly-Arg peptides), 3,4 proteins (e.g., antibodies,5 transport proteins, and transferrin6), and small molecules (e.g., folate7) have been used to selectively direct drugs to cancer cells with upregulated receptors by forming drug-carrier conjugates (Fig. 1). (biosyn.com)
  • The present invention envisions phosphorylated forms of any one of these GEF-H1 peptides, polypeptides and proteins. (justia.com)
  • The synthesized conjugate exhibits dose-dependent antisense activity. (nih.gov)
  • This conjugate will be designed to be stable in the upper gastrointestinal tract and deliver locally the nucleic acid drug to the colonic mucosa. (grstiftung.ch)
  • This review is focused on understanding the factors involved in the development of an effective drug-peptide conjugate with an emphasis on the chemistry and biology of the conjugates. (biosyn.com)
  • Synthetic antisense DNAs are used to hybridize to complementary sequences in target RNAs or DNAs to effect the functioning of specific genes for investigative or therapeutic purposes. (genomicglossaries.com)
  • They can bind to the desired complementary sequence, thereby, can target specific RNAs. (biologyonline.com)
  • The review also outlines the vast applications of UBPs as novel information storage components as well as in creation of semi-synthetic organisms expressing non canonical amino acids, high affinity aptamer generation, PCR based diagnostics and sitespecific labelling of RNAs. (fortuneonline.org)
  • Double-stranded nucleic acid molecules (DNA-DNA or DNA-RNA) which contain regions of nucleotide mismatches (non-complementary). (umassmed.edu)
  • The approaches and results presented here will serve as a guide to systematically approach targeted delivery of cytotoxic drug molecules using peptides for treatment of several diseases. (biosyn.com)
  • The specific characters of peptides (high bioactivity, high specificity, and low toxicity) have made them attractive therapeutic agents. (intechopen.com)
  • Peptide nucleic acids (PNAs) 1 are synthetic structural homologues of DNA and RNA in which the entire phosphate-sugar backbone of the polynucleotide has been replaced by a flexible polyamide backbone consisting of 2-aminoethyl glycine units. (archive.org)
  • We have now tested the effects of CAG-specific and c-mycspecific PNAs on AR, TBP, and c-myc transcription in human prostatic cancer cell lines, and we now show that each PNA can selectively target its complementary DNA sequence in the intact chromatin of permeabilized cells. (archive.org)
  • These advantageous features instigated tremendous interest in PNAs as antisense and antigene agents. (creative-peptides.com)
  • To date, four ASOs compounds have received a marketing authorisation and there are currently more than 100 clinical trials with antisense compounds. (antisenserna.eu)
  • 1. James, W. Towards gene-inhibition therapy: a review of progress and prospects in the field of antiviral antisense nucleic acids and ribozymes / W. James // Antiviral Chem .and Chemotherapy. (finechem-mirea.ru)
  • These investigations have resulted in reports of a partially purified 68 kilodalton (Kd) protease which cleaves at a Met-Asp bond of a synthetic peptide. (everypatent.com)
  • Then the synthetic peptide vaccine application of peptides was reviewed. (intechopen.com)
  • Following reconstitution of the human immune system, inhibition of TGF-ß signaling by TGF-ß2 antisense oligodeoxynucleotide (TASO) resulted in accelerated tumor growth inhibition. (bvsalud.org)
  • and Uversky, Vladimir N., "Intrinsic Disorder in Protein Sense-antisense Recognition" (2020). (usf.edu)
  • It thus also increases in vivo expression of a protein encoded by such a nucleic acid upon increased transfection rate. (patsnap.com)
  • The DNA sequence of the genes for the androgen receptor (AR) and TATA-binding protein (TBP), like many other genes encoding transcription factors, contains a series of tandem CAG repeats. (archive.org)
  • Intrinsic Disorder in Protein Sense-antisense Recognition" by Guy W. Dayhoff, Marc H. Regenmortel et al. (usf.edu)
  • This invention provides an APP-cleaving protein and related nucleic acid compounds. (everypatent.com)
  • To study these strains, we sequenced cDNA fragments of protein E gene from six archival formalin-fixed brain samples. (cdc.gov)
  • Analysis of the protein E sequence of 16 European and Asian TBEV strains showed clear segregation into three genetic subtypes, designated as European, Far Eastern, and Siberian ( 4 ). (cdc.gov)
  • To determine the TBEV genotype that probably caused the hemorrhagic form of the infection, we sequenced cDNA fragments of protein E gene. (cdc.gov)
  • All recognized mutations for AD are associated with increased deposition of amyloid-beta (Abeta), a peptide fragment comprising 39-43 amino acids that derive from the catabolism of the amyloid precursor protein (APP) molecule. (medscape.com)
  • In a preferred embodiment, a GEF-H1 polypeptide is provided that lacks the amino acid region between residues 162 and 354 of SEQ ID NO. 2. (justia.com)
  • 1. A purified protease which comprises the amino acid sequence SEQ ID NO:1, or a functional equivalent thereof produced by conservative amino acid alterations which result in a protease which exhibits the same biological, biochemical, chemical, physical and structural properties of the protease of SEQ ID NO:1. (everypatent.com)
  • vi)a nucleic acid sequence according to any of (i) and (ii) which is degenerate as a result of the genetic code. (allindianpatents.com)
  • We have shown in in vitro experiments that the active antisense structure is restored by reduction of azo-bonds between protective PEG chains and conjugate's lysine residues. (grstiftung.ch)
  • Conversely, a PNA complementary to a unique sequence of the c-myc gene did not inhibit transcription of the AR or TBP genes but did inhibit c-myc transcription. (archive.org)
  • The absence of phosphate groups in the PNA molecule facilitates its invasion of negatively charged DNA duplexes containing the complementary base sequences (4 -6). (archive.org)
  • When a PNA mol-ecule invades a targeted DNA sequence, one strand of the DNA is displaced (1, 5, 6, 9) , whereas the PNA binds quickly to its complementary DNA sequence by Watson-Crick base pairing (7). (archive.org)
  • Molecular biologists describing DNA sequences or referring to one of the two strands of double- stranded DNA frequently use complementary pairs of terms, such as coding/ non- coding, sense/ nonsense or transcribing/ non- transcribing. (genomicglossaries.com)
  • Oral delivery of nucleic acid drugs to the colon would be highly desirable to provide new options for the treatment of serious and rare colonic diseases such as ulcerative colitis, Crohn's colitis, and Lynch syndrome, but has been challenging due to the harsh conditions found in the gastrointestinal tract and low cellular uptake. (grstiftung.ch)
  • Treatments based on nucleic acids have, while conceptually promising, failed to meet initial expectations largely because of inadequate delivery to the target site and poor or inefficient cellular uptake. (grstiftung.ch)
  • Despite the advantageous features of classic PNA, its low water solubility, poor cellular uptake, propensity to self-aggregate, and orientational ambiguity in recognizing nucleic acid targets, limits the effectiveness of PNA in its applications. (creative-peptides.com)
  • In the last part, information about cell-penetrating peptides that can be used as molecular carries is mentioned with providing classification and cellular uptake mechanism of them. (intechopen.com)
  • A recent study using in vivo biochemical probing and comparative sequence analysis proposed a revision of the repA structure model that includes both intra-repeat and inter-repeat folding found in previous models as well as novel features (see Figure). (wikipedia.org)
  • Because of its antigene, antisense, anti-miR, and the scope to be labeled with various fluorophores, PNA finds application in gene expression modulation in-vivo , and development of nucleic acid-based biosensors which are useful in biotechnology researches and clinical diagnostics. (creative-peptides.com)
  • Cannon W, Gallegos MT, Casaz P, Buck M. Amino-terminal sequences of sigmaN (sigma54) inhibit RNA polymerase isomerization. (umassmed.edu)
  • Chemical modification or analogs of nucleic acids can increase resistance to enzymatic degradation but do not solve the challenges of delivery. (grstiftung.ch)
  • Peptide conjugated morpholinos for management of the huanglongbing pathosystem. (yale.edu)
  • It is best to be aware of these definitions while sticking with the recommendations of the Human Genome Variation Society and to use ' sequence variant ' , ' alteration ' or ' allelic variant ' for any genomic change regardless of their frequency or phenotypic effects. (tripod.com)
  • While the genomic sequence of DcAOX2a was previously described, we characterize here the complete genomic sequence of DcAOX1 . (frontiersin.org)
  • This work developed a rapid strategy for intracellular delivery of nucleic acids, thus providing more possibilities toward personalized cancer medicine. (bvsalud.org)
  • The present invention is directed to (the use of) a solution containing at least one nucleic acid (sequence) and free mannose for lyophilization, transfection and/or injection, particularly of RNA and mRNA. (patsnap.com)
  • The sense strand has the same sequence as the mRNA transcript. (genomicglossaries.com)
  • According to the National Centre for Biotechnology Information (NCBI), therapeutic nucleic acids (TNAs) are a subset of nucleic acids that are closely related compounds used to cure disease through the sequence-specific recognition of endogenous nucleic acids. (gii.tw)
  • The functional role of the Xist transcript was definitively demonstrated in mouse female ES cells using a novel antisense technology, called peptide nucleic acid (PNA) interference mapping. (wikipedia.org)
  • The chromatin-binding region was functionally mapped and evaluated by using an approach for studying noncoding RNA function in living cells called peptide nucleic acid (PNA) interference mapping. (wikipedia.org)
  • The present invention provides lipid-based formulations for delivering, e.g., introducing, nucleic acid-lipid particles comprising an interference RNA molecule to a cell, and assays for optimizing the delivery efficiency of such lipid-based formulations. (patsnap.com)
  • Alternatively spliced transcript variants have been identified, but their full length sequences have not been determined. (wikipedia.org)
  • We have shown previously that a biotinylated PNA targeted to CAG repeats will strand invade those DNA sequences in their transcriptionally active states in intact chromatin (21). (archive.org)
  • When referring to DNA, the terms should be "transcribed strand" and 'non- transcribed strand" The term antisense would be best reserved for RNA. (genomicglossaries.com)
  • When DNA sequences are described by giving the sequence of only one strand, this is usually the strand with the same sequence as the RNA (messenger, ribosomal, transfer, etc.) and should therefore be called the coding strand. (genomicglossaries.com)
  • 2. Sequence-selective recognition of DNA by strand displacement with a thymine-substituted polyamide / P. E Nielsen, M. Egholm, R. H. Berg, О. (finechem-mirea.ru)
  • At DARTER COST Action, we use networking and capacity building to allow RNA-targeting nucleic acid drugs to reach their full potential and become a mainstream therapeutic option. (antisenserna.eu)
  • Nucleic Acid Heteroduplexes" is a descriptor in the National Library of Medicine's controlled vocabulary thesaurus, MeSH (Medical Subject Headings) . (umassmed.edu)
  • The present invention is furthermore directed to a method of lyophilization using the mannose-containing solution, to pharmaceutical compositions, vaccines, kits, first and second medical uses applying such a mannose-containing solution and/or a nucleic acid (sequence) lyophilized or resuspended with such a solution. (patsnap.com)
  • Accordingly, the present invention envisions an isolated nucleic acid encoding the GEF-H1S polypeptide of SEQ ID NO:2, as well as an isolated nucleic acid comprising the sequence of SEQ ID NO:1. (justia.com)
  • In this context, the invention also contemplates an isolated GEF-H1S polypeptide comprising the sequence of SEQ ID NO:2. (justia.com)
  • The invention also envisages a peptide consisting essentially of the sequence of SEQ ID NO. 3. (justia.com)
  • The conformationally preorganized helical PNA has an excellent affinity and high sequence selectivity to bind the DNA and RNA. (creative-peptides.com)
  • In nucleic acids, sense-antisense complementarity is achieved via the Watson-Crick complementarity of the base pairs or nucleotide pairing. (usf.edu)
  • A nucleotide is regarded as the basic building block of nucleic acid polymers (e.g. (biologyonline.com)
  • In the reported experiments, a single 19-bp antisense cell-permeating PNA targeted against a particular region of Xist RNA prevented the formation of Xi and inhibited cis-silencing of X-linked genes. (wikipedia.org)
  • In the reported experiments, a single 19-bp antisense cell-permeating PNA targeted against a particular region of Xist RNA caused the disruption of the Xi. (wikipedia.org)
  • In parallel, this research allowed the identification of other local nucleic acid delivery strategies for the treatment of intestinal diseases. (grstiftung.ch)
  • The use of synthetic peptides approved by the health authorities for vaccine, for cancer, and in drug delivery systems is increasing with these developments. (intechopen.com)
  • They usually act through binding to specific sequences in coding RNA, so they are highly accurate . (antisenserna.eu)
  • In another embodiment, serine-67 is present in a peptide of less than 30 amino acids that comprises the sequence of SEQ ID NO. 4. (justia.com)
  • The aim of this book chapter is to review the recent developments in the use of peptides in the diagnosis of drug and vaccine systems and to present them to the reader with commercially available illustrations. (intechopen.com)
  • The inventive solution exhibits a positive effect on stabilization of the nucleic acid (sequence) during lyophilization and storage but also leads to a considerable increase of the transfection efficiency of a nucleic acid. (patsnap.com)
  • 10. McKennon, M. A convenient reduction of amino acids and their derivatives / M. McKennon, A. Meyers // J. Org. (finechem-mirea.ru)