Abstract: Polynucleotides and vectors can be used for the expression of a transgene in cells, such as liver cells. The expression of the transgene from the polynucleotides and vectors can be useful in gene therapy. Various methods can be used for expressing the transgene from the polynucleotides and vectors in liver cells.

Abstract: An orthopaedic device for securing between first and second regions of bone separated by a growth plate, the orthopaedic device comprising: a first portion to secure to a first fixing location on the first bone region; a second portion to secure to a second fixing location on the second bone region, the first fixing location and the second fixing location being laterally offset relative to a longitudinal axis of the bone; wherein the first and second portions are pivotally coupled between a first coupling location on the first portion and a second coupling location on the second portion such that movement of the first and second portions away from each other in a direction parallel to the longitudinal axis of the bone causes relative rotation of the first bone region and the second bone region thereby reducing the lateral offset of the first location and the second location.

Abstract: Provided herein are methods for alleviating symptoms in a subject having Rett syndrome, comprising administering to the subject an effective amount of an isolated binding molecule which specifically binds to semaphorin-4D (SEMA4D).

Abstract: The present invention relates to identification of an abnormal splice site. Provided are methods of identifying an abnormal splice site. Methods of classifying the risk of abnormal splicing of a splice site are also provided. Databases for use in the methods provided herein are also disclosed.

Abstract: The present disclosure relates generally to promoters derived from the AAV anti-sense strand, their use in the expression of one or more heterologous coding sequences, and isolated polynucleotides, vectors and recombinant viruses comprising the promoters. The present disclosure also relates to enhancers derived from the AAV anti-sense strand, their use in increasing the expression of one or more heterologous coding sequences, and isolated polynucleotides, vectors and recombinant viruses comprising the enhancers.

Abstract: The present disclosure relates generally to polypeptides derived from marsupial adeno-associated vims (AAV). The disclosure is also related to nucleic acid molecules encoding the polypeptides, and vectors comprising the nucleic acid molecules, and AAV vectors comprising the polypeptides. The disclosure also relates to uses of the nucleic acid molecules, polypeptides and AAV vectors, such as for capsid diversification.

Abstract: Polypeptides are encoded by an adeno-associated virus (AAV)-derived endogenous viral element (mAAV-EVE1) found within the germline of numerous closely-related marsupial species. Nucleic acid molecules encode the polypeptides. Vectors can include the nucleic acid molecules, and recombinant AAV virions can include the polypeptides. A chimeric capsid protein can also include an MAAV-EVE1 polypeptide.

Abstract: An intramedullary orthopaedic device is disclosed including an intramedullary rod configured for insertion into a bone, the rod including a first elongate member having a first mating surface and at least a second elongate member having a second mating surface. The first mating surface is configured to engage with the second mating surface such that the elongate members are longitudinally moveable but substantially not rotationally moveable relative to each other. Each mating surface can have an elongate ridge and an elongate groove. A guided growth system and method is also disclosed.

Abstract: Polynucleotides and vectors can be used for the expression of a transgene in cells, such as liver cells. The expression of the transgene from the polynucleotides and vectors can be useful in gene therapy. Various methods can be used for expressing the transgene from the polynucleotides and vectors in liver cells.

Abstract: Polypeptides are encoded by an adeno-associated virus (AAV)-derived endogenous viral element (mAAV-EVE1) found within the germline of numerous closely-related marsupial species. Nucleic acid molecules encode the polypeptides. Vectors can include the nucleic acid molecules, and recombinant AAV virions can include the polypeptides. A chimeric capsid protein can also include an MAAV-EVE1 polypeptide.

Abstract: A device and method for correcting a rotational deformity, for use in lengthening a bone or aiding in stabilising a fractured bone. When used to correct a rotational deformity, the device comprises a first member and a second member moveably connected to each other such that when the two members are moved apart along a first axis, the first and/or second members rotate relative to each other about the first axis.

Abstract: An orthopaedic device for correcting deformities in a bone is disclosed, the device including first and second end portions to fix to first and second bone regions either side of a growth plate, and a connection portion extending between the first and second end portions. At least the first end portion can protrude relative to the connection portion such that it is at least partly receivable in a first recess formed in the first bone region. The first end portion can be in the form of an end cup and can be fixed to the first bone region by a fixation device such as a screw. A head of the screw can sit within the end cup. The second end portion can be similar to the first end portion or alternatively may comprise a hook, blade or staple.

Abstract: A system (10) and method for use by a subject with a sleep disorder, such as nocturnal enuresis. The system (10) comprises at least one sensor (11) that detects at least one attribute of the sleep disorder, for example urine, and outputs at least a first signal on or after detection of that attribute. The system (10) further comprises a control device (12) that comprises an arousal device (14), such as a loudspeaker, that activates on or after receipt of the first signal, an interactive device (15) that is actuable by the subject to deactivate or modify the operation of the arousal device (14) and an output device (16) for delivering an output to the subject. The output being delivered on or after deactivation or modification of the arousal device (14).

Abstract: A system (10) and method for use by a subject with a sleep disorder, such as nocturnal enuresis. The system (10) comprises at least one sensor (11) that detects at least one attribute of the sleep disorder, for example urine, and outputs at least a first signal on or after detection of that attribute. The system (10) further comprises a control device (12) that comprises an arousal device (14), such as a loudspeaker, that activates on or after receipt of the first signal, an interactive device (15) that is actuable by the subject to deactivate or modify the operation of the arousal device (14) and an output device (16) for delivering an output to the subject. The output being delivered on or after deactivation or modification of the arousal device (14).