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This invention relates to a L-asparaginase enzyme variant based on the relevant gene from Lachancea thermotolerance modified by inducing a mutation at position Lys99. The enzyme in question exhibits very high specific activity when compared to the wild type enzyme and the enzyme in the known state of the technique. The enzyme in question is suitable for the treatment of acute lymphoblastic leukemia.

The present invention relates to a nucleic acid sequence encoding the transcription factor ESE3/EHF or an isoform thereof, for use in the treatment of a cancer in a patient in need thereof and can be a mRNA or a DNA, in particular for use in the treatment of prostate cancer. The invention also relates to a plasmid, a viral vector or a pharmaceutical composition comprising, such nucleic acid sequence. In particular, the present invention is based on the use of plasmid DNA coding the full-length sequence of the gene ESE3/EHF and to the use of in vitro transcribed (IVT) mRNA of ESE3/EHF as gene therapy in aggressive prostate cancerESE3/EHF is a transcription factor that is expressed in normal prostate but is lost in prostate tumors, particularly during the progression from indolent to aggressive tumors. The aim is to replace the ESE3/EHF transcription factor using gene therapy approaches at the time the gene is reduced or lost. The invention also relates to the use of ESE3/EHF replacement in combination therapy with androgen deprivation therapy (ADT), the standard treatment for metastatic prostate cancer, and other therapies, such as androgen receptor signalling inhibitors (ARSI), chemotherapeutics, molecular-targeted therapeutics, and immunotherapeutics.

A method of producing a three-dimensional synovium-on-chip (SoC), a three-dimensional synovium-on-chip as well as the respective uses in drug screening methods.

本发明公开了一种高纯度dsRNA的制备方法，本发明利用蛋白纯化平台，提供一种高纯度dsRNA的制备方法，主要包括菌体预处理，上样样品配制，I型阴离子柱收集流穿和吸附，II型阴离子柱收集吸附，切向流浓缩和置换。制备的dsRNA纯度高，收率高，得到的dsRNA纯度在90％以上，为dsRNA使用纯化平台制备提供了可靠方法。本发明缓冲液中使用钾盐，相较于钠盐，洗脱力更强；并且本发明的处理量更大，可以放大到300L的柱子，一次处理400kg菌体，甚至更大，有利于生产使用；本发明收率更高，是老工艺的8倍；使用的有机溶剂更少，节省成本而且环保。

Provided are compositions comprising an oligonucleotide that targets Angiopoietin-like 4 (ANGPTL4). The oligonucleotide may include a small interfering RNA (siRNA) or an antisense oligonucleotide (ASO). Also provided herein are methods of treating a metabolic or cardiovascular disorder by providing an oligonucleotide that targets ANGPTL4 to a subject in need thereof.

The present invention provides circular RNA structures with improved self-circularization efficiency and/or enhanced suppression of impurity generation through engineering. The self-circularizing RNA structure of the present invention can be used universally for various GOIs and is expected to be utilized in diverse biotechnology fields including vaccine development gene therapy products and protein expression systems aimed at high-efficiency circular RNA production

Disclosed herein are methods that may be used for the synthesis of benzylisoquinoline alkaloids ("BIAs") such as alkaloid morphinan. The methods disclosed can be used to produce thebaine, oripavine, codeine, morphine, oxycodone, hydrocodone, oxymorphone, hydromorphone, naltrexone, naloxone, hydroxycodeinone, neopinone, and/or buprenorphine. Compositions and organisms useful for the synthesis of BIAs, including thebaine synthesis polypeptides, purine permeases, and polynucleotides encoding the same, are provided.

The present disclosure provides a novel transcriptional regulatory element comprising a mammalian endogenous promoter sequence and an enhancer element optionally operably linked to a mammalian intron sequence, thereby providing a potent fusion promoter for constant high levels of recombinant gene transcription in mammalian cells, including CHO cells or CHO-derived cells.

Provided are polynucleotides for expressing transposases, in particular, polynucleotide mRNAs for expressing piggyBac transposases comprising a piggyBac transposase coding sequence comprising hyperactive mutations and modified 5'- and 3'-UTR sequences to enhance piggyBac transposase expression, integration/excision activity and/or reduce in vivo immunogenicity of the encoded transposase.

"MULTIMERIC PROTEIN STRUCTURE AND PROCESS FOR PREPARING THE MULTIMERIC PROTEIN STRUCTURE". This document discloses multimeric protein structures comprising at least two alpha-galactosidase monomers covalently linked to each other via a linking group, as well as a process for preparing said structures, and methods for treating Fabry disease by administering a multimeric protein structure; the disclosed multimeric protein structures exhibit improved performance in terms of enhanced activity and/or longer-lasting activity under lososomal conditions and a serum environment.