The disclosure provides compositions and methods for the preparation, manufacture and therapeutic use of viral vectors, such as adeno-associated virus (AAV) particles having viral genomes encoding one or more antibodies or antibody fragments or antibody-like polypeptides, for the prevention and/or treatment of diseases and/or disorders.

The present invention provides an isolated nucleic acid molecule comprising, or consisting of, the nucleic acid sequence of SEQ ID NO:1 or a nucleic acid sequence of at least 300 bp having at least 80% identity to said sequence of SEQ ID NO:1, wherein said isolated nucleic acid molecule specifically leads to the expression in rod photoreceptors of a gene when operatively linked to a nucleic acid sequence coding for said gene.

Methods and materials for effective dosages of AAV gene therapy for the treatment and prophylaxis of hemophilia A.

The invention relates to a kit of parts comprising (i) pharmacological chaperones or a pharmaceutically acceptable salt thereof and (ii) a therapeutic acid-alpha glucosidase (GAA) polypeptide or a nucleic acid molecule encoding a therapeutic GAA polypeptide, wherein said pharmacological chaperones are 1-deoxynojirimycin (DNJ) or a derivative thereof and ambroxol (ABX) or a derivative thereof.

The invention provides genetic constructs and recombinant vectors comprising such constructs. The constructs and vectors can be used in gene therapy methods for treating a range of disorders, including glaucoma and deafness, or for promoting nerve regeneration and/or survival.

The present invention relates to a preparation method for an animal model with Alzheimer's disease by injecting a human mutant tau (AAV-hTau) vector and adenovirus into an animal. The preparation method for an AD animal model provided by the present invention may contribute to the development of the field of treatment technology for treating AD since the preparation method causes AD pathology to appear as early as 8 months old and facilitates studies on AD target treatment strategies and tau pathology.

Described are recombinant nucleic acid molecules for increased expression of Cas9 in human liver. In some embodiments, the recombinant nucleic acid molecules are provided in compositions and methods for gene editing, specifically using Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR).

Described herein are constructs used for liver-specific expression of a transgene.

Non-human animal cells and non-human animals comprising CRISPR/Cas synergistic activation mediator system components and methods of making and using such non-human animal cells and non-human animals are provided. Methods are provided for using such non-human animals to increase expression of target genes in vivo and to assess CRISPR/Cas synergistic activation mediator systems for the ability to increase expression of target genes in vivo.

Disclosed are methods for performing transcription-dependent directed evolution (TRADE) and novel AAV capsids selected using such methods.