Abstract: It is provided an electronic smoking device (10) which comprises a liquid reservoir (34), a battery (18) and a heating element (28) adapted to atomize liquid of the liquid reservoir (34). The heating element (28) has a modified surface (50) that comprises a plurality of structures (51) adapted to provide a capillary force on liquid of the liquid reservoir (34) when applied onto the heating element (28).

Abstract: The present invention relates to an adeno-associated vims (AAV) or an adeno-associated virus-like particle (AAVLP), comprising an insert of about 75-400 amino acids in the viral proteins (VPs) VP1, VP2 and/or VP3 at an insertion site (I) at the top of variable region VIII and/or variable region IV (VR-VIII and/or VR-IV) of the VP, wherein the insert is an immunogenic protein or a portion thereof and/or wherein the insert is a protein comprising a binding domain, such as an antigen-binding domain specific for a target antigen. The present invention also relates to pharmaceutical compositions comprising said AAV or AAVLP and to the pharmaceutical composition or the AAV or AAVLP for use in therapy, particularly for use as a vaccine, for use in the treatment or the prevention of a diseases and/or for use in gene therapy. Also concerned is a method for producing the AAV of AAVLP of the present invention.

Abstract: The present invention relates to a method of trans-activating a homologous gene of at least one gene of interest and optionally deactivation of at least one gene of interest, wherein the mRNA encoded by the at least one gene of interest comprises a mutation compared to a control, and wherein the method comprises the steps as described in the present application. The present invention further relates to an in vitro method of diagnosing a disease, wherein the method comprises the steps of: a) Inducing the expression of the mRNA encoded by at least one gene of interest in a cell or tissue sample obtained from a subject; b) isolating the mRNA of step a); c) analyzing the sequence of the isolated mRNA of step b) and d) thereby detecting a mutation of the mRNA compared to a control, which is indicative for the presence of the disease.

Abstract: The present invention relates to a polynucleotide configured for the treatment of a retinal degeneration disease, such as retinitis pigmentosa (RP), a nucleic acid vector comprising said polynucleotide, a pharmaceutical composition comprising said nucleic acid vector, a kit comprising said polynucleotide or said nucleic acid vector, a method of making said nucleic acid vector, and a method for treating a retinal degeneration disease.

Abstract: The present invention relates to an acceptor splice region, as well as uses and applications thereof.

Abstract: The present invention relates to a polynucleotide configured for the treatment of a disease of retinal cone cells, such as achromatopsia, a nucleic acid vector comprising said polynucleotide, a pharmaceutical composition comprising said nucleic acid vector, a kit comprising said polynucleotide or said nucleic acid vector, a method of making said nucleic acid vector, and a method for treating a disease of the retinal cone cells.

Abstract: The present invention relates to an adeno-associated virus (AAV), comprising an insertion of at least 6-8 amino acids between the positions corresponding to position 587 and 588 of SEQ ID NO: 1. Also envisioned are AAVs of the present invention for use as a medicament and pharmaceutical compositions comprising the AAV of the present invention. Further, the present invention relates to an in vitro use of AAV of the present invention for transduction of the nucleus of retinal cells. Also concerned is a method for screening an insertion sequence as well as a peptide obtainable by the method for screening. Also contemplated are kits comprising the AAV of the present invention.

Abstract: The present invention relates to a polynucleotide comprising a promoter comprising a human photoreceptor-specific promoter element, a core promoter and at least one transgene. Further, the invention provides a plasmid comprising the polynucleotide, a viral vector comprising the polynucleotide and a pharmaceutical composition comprising the polynucleotide. The invention also relates to the plasmid, the viral vector or the pharmaceutical composition for use as a medicament, in particular for use in the therapy of diseases of the retina.

Abstract: The present invention relates to a polynucleotide configured for the treatment of a retinal degeneration disease, such as retinitis pigmentosa (RP), a nucleic acid vector comprising said polynucleotide, a pharmaceutical composition comprising said nucleic acid vector, a kit comprising said polynucleotide or said nucleic acid vector, a method of making said nucleic acid vector, and a method for treating a retinal degeneration disease.

Abstract: The present invention relates to a polynucleotide configured for the treatment of a disease of retinal cone cells, such as achromatopsia, a nucleic acid vector comprising said polynucleotide, a pharmaceutical composition comprising said nucleic acid vector, a kit comprising said polynucleotide or said nucleic acid vector, a method of making said nucleic acid vector, and a method for treating a disease of the retinal cone cells.