Producing adenovirus gene therapy vector in producer cells that express or over-express adenoviral polypeptide IX enables one to produce pIX-deleted adenovirus in suspension cell culture. Using producer cells that express or over-express adenoviral polypeptide IX also increases the yield of adenovirus vector, regardless of whether that adenovirus is pIX-deleted. Using producer cells that express or over-express adenoviral polypeptide IX also improves the resulting vector's transduction kinetics, reducing the number of pfu/target cell required to achieve a given level of transduction/infection, shortening the time the vector requires to transduce or infect a target cell, and shortening the time an infected target cell produces progeny virus.

Novel forward primer, reverse primer and poly-linker suitable for replication of nucleic acids in e.g., 293 cells.

An object of the present invention is to provide a method for determining whether a subject suffers from malignant lymphoma or leukemia and an agent for treating and/or preventing the disease. The present invention relates to a method for assisting in determining whether a subject suffers from, or is likely to suffer from malignant lymphoma or leukemia, comprising: a detection step of detecting at least one of a fusion mutation of a DUX4 gene, an overexpression of a DUX4 gene, and a fusion mutation of an MEF2D gene; and a determination step of determining that the subject suffers from or is likely to suffer from the disease when at least one of the fusion mutations or the overexpression is detected. Moreover, the present invention relates to a pharmaceutical composition comprising a DUX4 inhibitor as an active ingredient, for treating and/or preventing malignant lymphoma or leukemia in a subject having a fusion mutation of a DUX4 gene and an IGH or IGL gene and/or overexpression of a DUX4 gene.

Lipid-based vesicles, typically herein called transfection competent vesicles (TCVs), configured to safely and efficiently deliver DNA, RNA, other nucleic acid and protein cargoes into target cells. The safety and efficiency are each, and both, achieved in part by eliminating organic solvents such as ethanol and detergents such as sodium dodecyl sulfate from the TCV loading processes (i.e., inserting a cargo into the TCV), TCV storage processes, and/or TCV delivery processes. The cargoes can also comprise nucleic acids complexed with a protein, such as a ribonucleoprotein (RNP). The systems, compositions, devices and methods, etc., herein, in some embodiments, can provide empty TCVs that can if desired be loaded at the bench without use of specialized equipment.

The invention is directed to a recombinant Adeno-Associated Virus (rAAV) vector particle having at least one primary amino group contained in the capsid proteins, chemically coupled with at least one ligand L, wherein coupling of said ligand L is implemented through a bond comprising a CSNH bond and an optionally substituted aromatic moiety. The present invention further relates to a method for chemically coupling an Adeno-Associated Virus (AAV) vector particle with at least one ligand L and to a Recombinant Adeno-Associated Virus (rAAV) vector particle obtained by said method as well as a pharmaceutical composition comprising it and their corresponding medical use.

Aspects of the disclosure relate to liquid chromatography (e.g., HPLC) methods which enable high resolution separations of polynucleotides of various lengths, sequences, and/or base compositions in a highly tunable manner. In some embodiments, the disclosure describes liquid chromatographic methods for separating a nucleic acid (e.g., a polyadenylated nucleic acid, such as an mRNA) from a complex mixture by using multiple ion pairing agents in the same mobile phase system. Accordingly, in some embodiments methods described by the disclosure are useful for assessing the quality of pharmaceutical preparations comprising nucleic acids.

Disclosed herein are compositions and methods for modulating the production of a protein in a target cell. The compositions and methods disclosed herein are capable of ameliorating diseases associated with protein or enzyme deficiencies.

A method of preparing a therapeutic cell population for clinical use from a starting population of cells comprising haematopoietic stem cells, said method comprising separating a population of cells that substantially do not express CD38 but which express CD34 from the starting population of cells, and transducing the separated cell population with a vector to obtain the therapeutic cell population.

A recombinant adeno-associated virus (rAAV) particle with a mutated capsid protein is provided. In particular, the present disclosure provides methods of delivering a therapeutic agent to a muscle, airway, liver, central nervous system, retina or lung cell in a subject, and methods of treating or preventing infectious, acquired or genetic disease, with said rAAV particle.

A method of correcting singletons in a selected AAV sequence in order to increasing the packaging yield, transduction efficiency, and/or gene transfer efficiency of the selected AAV is provided. This method involves altering one or more singletons in the parental AAV capsid to conform the singleton to the amino acid in the corresponding position(s) of the aligned functional AAV capsid sequences.