Abstract: The present invention relates to conjugates formed from a cell-penetrating peptide carrier linked to a therapeutic molecule, wherein the peptide carrier is defined by specific domains and the therapeutic molecule is a nucleic acid formed of trinucleotide repeats. The present invention further relates to the use of such a conjugate in methods of treatment or as a medicament, especially in the treatment of trinucleotide repeat disorders such as myotonic dystrophy (DM1).

Abstract: The invention relates to a recombinant adeno-associated virus (AAV) capsid protein, which is a peptide-modified hybrid between AAV serotype 9 (AAV9) and AAV serotype 74 (AAVrh74) capsid proteins comprising at least one copy of a peptide comprising the RGD motif, wherein said recombinant peptide-modified hybrid AAV capsid protein has a further reduced liver tropism and an increased muscle transduction compared to the recombinant hybrid AAV capsid protein not having said peptide. The invention relates also to the derived peptide-modified hybrid AAV serotype vector particles packaging a gene of interest and their use in gene therapy, in particular for treating neuromuscular genetic diseases, in particular muscular genetic diseases.

Abstract: The present invention relates to a method for removing undesired anti-AAV antibodies from a blood-derived composition.

Abstract: A device for measuring a force, includes: a fastening apparatus; and a sensor, which includes a measuring member arranged to move along a measurement axis, the sensor being arranged to measure a force exerted on the measuring member depending on the movement of this measuring member. The fastening apparatus is arranged to exert, during a movement of the measuring member along this measurement axis, on a carrier to which they are fastened, an average force borne by the measurement axis.

Abstract: The invention relates to a recombinant adeno-associated virus (AAV) capsid protein, which is a hybrid between AAV serotype 9 (AAV9) and AAV serotype 74 (AAVrh74) capsid proteins, wherein said recombinant hybrid AAV capsid protein has a reduced liver tropism compared to the parent AAV9 and AAVrh74 capsid proteins. The invention relates also to the derived hybrid AAV serotype vector particles packaging a gene of interest and their use in gene therapy, in particular for treating neuromuscular genetic diseases.

Abstract: The present invention relates to a method for estimating the physical activity exerted by an upper limb (10) of a person (1), the method being characterised in that it comprises the steps of: (a) Acquiring, by inertial measurement means (20) solidly attached to a forearm (11) of said upper limb (10) of said person (1), an angular speed of said forearm (11); (b) Estimating, by data processing means (21, 31, 41), a torque exerted by the muscles of an arm (12) of the upper limb (1) on said forearm (11) as a function of the measured angular speeds, of an orientation of said forearm (11), and of physical parameters of said forearm (11); (c) Determining, by the data processing means (21, 31, 41), a power exerted by the upper limb (10) as a function of the estimated torque and of the measured angular speed.

Abstract: The invention relates to an allele specific siRNA able to silence the expression of only one allele of a heterozygous DNM2 gene, for treating diseases caused by heterozygous mutation and/or overexpression of Dynamin 2.

Abstract: The present invention relates to compositions and methods for treating myotonic dystrophy.

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

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

Abstract: The present invention relates to an animal model of myasthenia gravis, and to uses thereof.

Abstract: A method for locating a mobile element in a predetermined zone, including supplying power to an on-board module in the mobile element, where the on-board module includes an electronic circuit and an on-board coil, generating a locating signal by the electronic circuit and transmission of the locating signal via the on-board coil, picking up the locating signal by receiver coils on a support in proximity to the predetermined zone, each of the receiver coils configured to pick up the locating signal when the mobile element is in proximity, and determining a location of the mobile element in the predetermined zone by detecting a signal level on the support in the form of an array by a processing unit connected to the support. The electronic circuit and the on-board coil constitute an RLC circuit that oscillates, generating the locating signal by sudden interruption of the current through the on-board coil.

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: 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 provided methods and compositions for generating novel nucleic acid molecules through targeted spliceosome mediated simple or double trans-splicing. The compositions of the invention include pre-trans-splicing molecules (PTMs) designed to interact with a target precursor messenger RNA molecule (target pre-mRNA) and to mediate a simple or double trans-splicing reaction resulting in the generation of a novel chimeric RNA molecule (Chimeric RNA).

Abstract: The invention relates to a device for measuring the pinch force between a person's thumb and index finger, comprising: first and second parallel blades, said blades being disposed at a distance from one another and embedded at a first longitudinal end thereof; a sensor for measuring the pinch force exerted between the two blades at the second longitudinal end thereof, perpendicularly to the respective main planes thereof; a means for displaying the pinch force; and a means for storing and/or processing the pinch force. According to the invention, the first blade forms part of a rigid parallelepiped frame and the second blade is disposed in the space inside the parallelogram.

Abstract: The present invention relates to compositions and methods, in particular to methods based on systemic administration of scAAV, for delivering genes to cells of the retina of mammals, and in particular to photoreceptor cells, ganglion cells, glial cells, inner nuclear layer cells or cells of the retinal pigmented epithelium.