Abstract: A composition comprising a gene therapy product for use in the treatment of a dystrophic disease in a subject, advantageously in humans, wherein: the gene therapy product comprises a nucleic acid sequence encoding a functional microdystrophin; the composition is systemically administered.

Abstract: A composition comprising a gene therapy product for use in the treatment of a dystrophic disease in a subject, advantageously in humans, wherein: —the gene therapy product comprises a nucleic acid sequence encoding a functional microdystrophin; —the composition is systemically administered.

Abstract: The present invention relates to nucleic acids, compositions and methods for the treatment of diseases, in particular for the treatment of facioscapulohumeral dystrophy.

Abstract: The present invention relates to the combined use of antisense oligonucleotides and viral vectors for the treatment of Duchenne muscular dystrophy.

Abstract: A tube for chemical, biological or biotechnological matter is provided, wherein the tube has a positioning element, the positioning element shaped for engagement in a corresponding structure provided in a carrier.

Abstract: A gripper unit for handling a vessel for receiving biological material is proposed, inter alia. The vessel has a lid which can assume an open position and a closed position. The gripper unit comprises a gripper for gripping and releasing the vessel, and a lid holder, for holding a lid in a defined position in relation to the vessel. The defined position is an open position of the lid.

Abstract: The present invention relates to nucleic acids, compositions and methods for the treatment of facioscapulohumeral dystrophy.

Abstract: The present invention relates to improved methods for processing fluids and to a fluid processing device (1) for use in a centrifuge comprising: (a) a first holder (14) form-fit to the shape of a first tube (18) for holding said first tube (18) whereby said first tube (18) has a first cross section (A1); and (b) a second holder (22) form-fit to the shape of a second tube (26) for holding said second tube (26) whereby said second tube (26) has a second cross section (A2) that is different from said first cross section (A1). With the fluid processing devices and the methods according to the invention, it is possible to simplify the centrifugal processing steps for a given fluid processing sequence and to automate them.

Abstract: Provided are tricyclo-DNA (tc-DNA) AON and methods employing tc-DNA AON for modifying splicing events that occur during pre-mRNA processing. Tricyclo-DNA (tc-DNA) AON are described that may be used to facilitate exon skipping or to mask intronic silencer sequences and/or terminal stein-loop sequences during pre-mRNA processing and to target RNase-mediated destruction of processed mRNA.

Abstract: Fluid processing tube for use in optical analysis comprising at least one first portion being made from a first material suitable for optical analysis and being configured to include two optical paths of different lengths, and at least one second portion connected to said first portion and being made from a second material different from said first material.

Abstract: The present invention relates to a nucleic acid molecule containing a sequence of tricyclo nucleosides joined by internucleoside phosphorothioate linkage. The invention also relates to synthetic antisense oligonucleotides and to methods employing the same.

Abstract: The present invention relates to platelet-shaped zinc-magnesium pigments, wherein the platelet-shaped zinc-magnesium pigments comprise the 40.8 to 67.8 mol % of zinc, 32.2 to 59.2 mol % of magnesium and 0 to 7 mol % of Mn, Li, Be, Y, Sn, Al, Ti, Fe, Cu and mixtures thereof, based in each case on the total molar amount of the elements Zn, Mg, Mn, Be, Y, Li, Sn, Al, Ti, Fe and Cu, where the molar percentages add up to 100 mol %, and the median thickness h50 of the pigments is less than 1 ?m. The invention further relates to the use and production of these pigments.

Abstract: A composition comprising a gene therapy product for use in the treatment of a dystrophic disease in a subject, advantageously in humans, wherein:—the gene therapy product comprises a nucleic acid sequence encoding a functional microdystrophin;—the composition is systemically administered.

Abstract: The invention relates to a method for the treatment of amyotrophic lateral sclerosis (ALS). Specifically, the invention implements the use of an antisense sequence adapted to affect alternative splicing in a human SOD1 pre-mRNA, thereby leading to the destruction of the skipped m RNA by the cell machinery.

Abstract: An isolated linear nucleic acid molecule comprising in this order: a first adeno-associated virus (AAV) inverted terminal repeat (ITR), a nucleotide sequence of interest and a second AAV ITR, wherein said nucleic acid molecule is devoid of AAV capsid protein coding sequences. The said nucleic acid molecule can be applied to a host at repetition without eliciting an immune response. Methods for producing and purifying this nucleic acid molecule, and use of the same for therapeutic purposes are also provided.

Abstract: Methods for increasing the efficiency of target tissue penetration of an adeno-associated virus (AAV) vector in a patient are provided. In some aspects, the methods involve inhibiting the interaction of the serum protein galectin 3 binding protein (G3BP) with AAV vector. Further provided are methods for reducing tissue distribution of a virus or for neutralizing a virus harbored by an organ destined for transplant, or newly transplanted, by administering a composition comprising G3BP.

Abstract: The present invention relates to improved compositions and methods for delivering and expressing therapeutic genes in mammals. More particularly, the invention stems from the unexpected discovery that a remarkable, massive and widespread therapeutic gene delivery and expression is obtained in mammals when a therapeutic gene is incorporated in a viral vector and administered both into the CSF and into the blood of the mammal. Such a combined administration leads to a surprising and substantial therapeutic benefit in the mammal as compared to administration in one single site, and tether enables the use of reduced doses of the virus. The invention may be used in any mammal, including human subjects, and is particularly suited to treat multi-systemic diseases, such as motor neuron or lysosomal disorders, where widespread expression of a therapeutic gene is desirable.

Abstract: The present invention relates to a gene therapy vector which is useful in the treatment or prevention of hypertrophic cardiomyopathy in a subject in need thereof. The gene therapy vector of the invention comprises a nucleic acid sequence encoding a cardiac sarcomeric protein and a cardiomyocyte-specific promoter which is operably linked to said nucleic acid sequence. The invention furthermore relates to a cell which comprises the gene therapy vector. Pharmaceutical compositions which comprise the gene therapy vector and/or a cell comprising said vector are also provided. In another aspect, the invention relates to a method for treating or preventing hypertrophic cardiomyopathy in a subject by introducing the gene therapy vector of the invention into a subject in need of treatment.

Abstract: The invention relates to a sample container, comprising a housing which forms a sample space for receiving a sample and has at least one circular opening which extends in a channel-shaped manner into the sample space, and further comprising a spherical closing element, wherein the diameter of the closing element only exceeds the diameter of the opening channel in at least one (closing) portion to such an extent that the closing element can be fixed in a force-locked manner by its largest circumference in the closing portion, wherein the spherical closing element is in contact with the housing, and the opening channel between the closing portion and the inner opening forms a protrusion which reduces the opening cross section of the opening channel with respect to the opening cross section in the closing portion.

Abstract: A tube for chemical, biological or biotechnological matter is provided, wherein the tube has a positioning element, the positioning element shaped for engagement in a corresponding structure provided in a carrier.