Tag: SRP

By Alex Philippidis At the start of 2023, the website for Pfizer CenterOne posted an article titled, “What Trends Will Shape the Pharma and Biotech Industries in 2023?” It quoted Rick L. Knight, the company’s global head of strategic account management, as follows: “We can continue to expect to see…

While a Phase 3 clinical trial of Elevidys (delandistrogene moxeparvovec-rokl) has failed to meet its main goal, the results nonetheless showed that, compared with a placebo, the approved gene therapy led to notable improvements across numerous measures of physical function in boys with Duchenne muscular dystrophy (DMD). Top-line findings from that…

Months after the FDA approved Sarepta Therapeutics’ gene therapy SRP-9001 (Elevidys) for the treatment of ambulatory patients with Duchenne muscular dystrophy (DMD), the company has announced topline data from the phase 3 EMBARK study (NCT05096221), a trial aimed to reinforce the agent’s efficacy. Full results from EMBARK are expected to…

Entry BLA55_00535       CDS       T05055                                  Name (GenBank) signal recognition particle-docking protein FtsY   KO K03110   fused signal recognition particle receptor Organism mpul  Mycoplasmopsis pullorum Pathway mpul02024   Quorum sensing mpul03060   Protein export mpul03070   Bacterial secretion system Brite KEGG Orthology (KO) [BR:mpul00001] 09120 Genetic Information Processing  09123 Folding, sorting and degradation   03060 Protein export    BLA55_00535 09130 Environmental Information Processing  09131 Membrane transport   03070 Bacterial…

Introduction Pulmonary infection is one of the most common diseases of the respiratory system, and severe infections in patients with underlying diseases are associated with a high mortality rate and substantial damage to human health. The rate of detecting bacteria and fungi by traditional culture is 21.7%.1 Identifying pathogenic bacteria…

Sarepta Therapeutics, Inc. has announced U.S. Food and Drug Administration (FDA) accelerated approval of ELEVIDYS (delandistrogene moxeparvovec-rokl), an adeno-associated virus(AAV)-based gene therapy for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene.  This indication is…

Sarepta Therapeutics today revealed that FDA has approved delandistrogene moxeparvovec-rokl, the first gene therapy to treat Duchenne muscular dystrophy (DMD). The company described the approval as a “watershed moment” in the history of treatment Duchenne in a statement announcing the approval. The therapy, to be marketed as Elevidys, is approved…

While life sciences investment has retreated from its all-time highs during the COVID-19 pandemic, investors surveyed by a national business law firm are still bullish about the sector based on the growth potential seen in artificial intelligence (AI), cell and gene therapy (CGT), and other new technologies. According to the…

In the U.S., only a limited number of patients will enter Sarepta Therapeutics‘ ENVISION, a Phase 3 study that’s part of the company’s strategy to gain global approval of SRP-9001 (delandistrogene moxeparvovec) as a gene therapy for Duchenne muscular dystrophy (DMD). These DMD patients will be non-ambulatory, or unable to walk….

RNA-targeted NBTs that are currently being used to upregulate gene expression address both transcriptional and translational mechanisms and can be roughly divided into two groups: (1) NBTs that increase mRNA abundance by enhancing transcription or increasing mRNA stability (Fig. 3) and (2) NBTs that optimize translation (Fig. 4). Strategies in the first…

Garnham PCC. Malaria parasites and other haemosporidia. London: Blackwell Scientific; 1966. Google Scholar  Perkins SL. Malaria’s many mates: past, present, and future of the systematics of the order Haemosporida. J Parasitol. 2014;100:11–25. Article  PubMed  Google Scholar  Perkins SL, Schaer J. A modern menagerie of mammalian malaria. Trends Parasitol. 2016;32:772–82. Article …

Entry 120500365         CDS       T07277                                  Symbol SRP54 Name (RefSeq) signal recognition particle 54 kDa protein   KO K03106   signal recognition particle subunit SRP54 [EC:3.6.5.4] Organism pmoa  Passer montanus (Eurasian tree sparrow) Pathway pmoa03060   Protein export Brite KEGG Orthology (KO) [BR:pmoa00001] 09120 Genetic Information Processing  09123 Folding, sorting and degradation   03060 Protein export    120500365 (SRP54) 09180 Brite Hierarchies  09183 Protein families: signaling…

The sellside expects a bumper year for blockbuster approvals, a recent Evaluate Vantage analysis revealed, but which products are creating all the excitement? A handful of significant new agents have already been approved, in Alzheimer’s disease, geographic atrophy and respiratory syncytial virus, and there is no shortage of pending regulatory…

Why 0/0 genotype refers to SNP and ALT != REF (in vcf file after freebayes) 1 Good morning, Could you please explain me, why 0/0 genotypes in vcf are SNPs? According to this description of GT: 0/0 the sample is a homozygous reference 0/1 the sample is heterozygous (carries both…

SINP HOME   HD-RNAS Homepage Search Database Advanced Search Options Non-redundant Dataset HD-RNAS Tutorial Download all sequences      Get help on search Enter Organism Name: (e.g., H. sapiens or Homo sapiens) Select Functional RNA Type: (For class tRNA and rRNA, select the radiobutton first.) Transfer RNA (tRNA)     ———AlylArginylAsparginylAspartylCysteinylGlycylGlutaminylGlutamylHistidylIsoleucylLeucylLysylMethionylPhenylalanylProlylSerylThreonylTryptophanylTyrosylValyl Ribosomal RNA (rRNA)    …

Systematic augmentation of GWAS with network propagation Recent studies have shown that a comprehensive protein interaction network is critical for network propagation efforts9. Here, we combined the International Molecular Exchange physical protein interaction dataset19 from IntAct (protein–protein interactions)20, Reactome (pathways)21 and SIGNOR (directed signaling pathways)22. To facilitate re-use of these…

SINP HOME   HD-RNAS Homepage Search Database Advanced Search Options Non-redundant Dataset HD-RNAS Tutorial Download all sequences Search By Organism Name: Enter Organism name: (help) (e.g., H. sapiens or Homo sapiens)         Search By Functional RNA Type: (For class tRNA and rRNA, select the radiobutton first.) (help) Transfer RNA…

FEB230803 (W) Collin Kelly, Jackson Lanzing, Tochi Onyebuchi (A) Carlos Magno (CA) Steve McNiven THE REVOLUTION STARTS HERE: COLD WAR – PART ONE! Bucky Barnes is the Outer Circle’s New Revolution, and he has enacted his deadliest move yet – an alliance with Sam Wilson’s nemesis, the White Wolf….

Cesarman E, Chang Y, Moore PS, Said JW, Knowles DM. Kaposi’s sarcoma-associated herpesvirus-like DNA sequences in AIDS-related body-cavity-based lymphomas. N Engl J Med. 1995;332:1186–91. Article  CAS  PubMed  Google Scholar  Nador RG, Cesarman E, Chadburn A, Dawson DB, Ansari MQ, Sald J, et al. Primary effusion lymphoma: a distinct clinicopathologic entity…

PRESS RELEASE Published February 9, 2023 Las Vega (Nevada), United States //— As per DelveInsight’s assessment, globally, Duchenne muscular dystrophy pipeline constitutes 75+ key companies continuously working towards developing 75+ Duchenne muscular dystrophy treatment therapies, analysis of Clinical Trials, Therapies, Mechanism of Action, Route of Administration, and Developments analyzes DelveInsight….

Differential expression of complement proteins in the different types of IM To define the transcriptomic profiles of patients with different types of IM, we performed bulk RNAseq on muscle biopsies from 132 IM patients, including those with DM (n = 44), AS (n = 18), IMNM (n = 54), and IBM (n = 16). The DM group included…

Genome assembly and chromosome identification A Plantago ovata genome reference was generated by utilizing a total of 5.98 M (7 cells, 40.21 Gb, N50 = 10.45 Kb, 50 bp–121.17 Kb) PacBio long reads and 636.5 million (47.74 Gb) Hi-C short-reads. PacBio reads were used to assemble contigs, while Hi-C reads were used to achieve chromosome-level assembly. The final…

Hello to all I am trying to run bedtools intersect with vcf file and a bed file (my goal is to add the depth data to my VCF) I get an error running this command: bedtools intersect -a depth.bed -b fish.vcf -wa -wb > $out The error: “Error: Invalid record…

RNAcentral is a database of non-coding RNA sequences that aggregates ncRNA data from over 40 member resources known as Expert Databases.1 Non-coding RNAs Similar to mRNAs, non-coding RNAs (ncRNAs) are transcribed from DNA but are not translated into proteins. NcRNAs are found in all organisms and have a broad range…

Hey, I am looking for a way to add samples ID names to the FORMAT in my vcf file. I have 10 sorted Bam files. I used Freebayes to create vcf files and my next step is merging all 10 files for VcfSampleCompare. And for that I need to define…

VCF Filter On Small Genomes 0 Hi guys, I am working on a yeast species (Candida glabrata) NGS data to find any mutations related to drug resistance. I am new in bioinformatics so I am using Galaxy.eu to get use to algorithms. There is literature about some genes that mutations…