Abstract: Disclosed are recombinant adenoviral compositions and methods for their use in treating disorders associated with epithelial tissues.

Abstract: The present invention provides recombinant adenoviral compositions and methods for their use in treating disorders associated with epithelial tissues.

Abstract: The invention relates to the in vivo transduction of hematopoietic stem and progenitor cells (HSPCs) in a subject, such as a human subject, and to the treatment of subjects suffering from various pathologies, such as blood diseases, metabolic disorders, cancers, and autoimmune diseases, among others.

Abstract: The current disclosure provides recombinant adenoviral vectors and adenoviral genomes that can accommodate or that contain a large transposon payload, for instance a transposon payload of up to 40 kb. The adenoviral vectors and genomes can deliver the large transposon payload into a target genome, for instance for gene therapy.

Abstract: The present disclosure provides, among other things, immune suppression regimens for in vivo gene therapy and uses thereof. In various embodiments of the present disclosure, in vivo gene therapy includes delivery of at least one exogenous coding nucleic acid sequence to a stem cell of the subject. Success of in vivo gene therapy can be inhibited or reduced by immunotoxicity. The present disclosure provides compositions and methods, including among other things immune suppression regimens, that reduce immunotoxicity of in vivo gene therapy, e.g., in vivo gene therapy including administration of a viral gene therapy vector to a subject.

Abstract: Systems and methods to selectively protect therapeutic cells by reducing CD33 expression in the therapeutic cells using base editors and targeting non-therapeutic cells with an anti-CD33 therapy are described. The selective protection results in the enrichment of the therapeutic cells while simultaneously targeting any diseased, malignant and/or non-therapeutic CD33 expressing cells within a subject.

Abstract: The present disclosure provides, among other things, helper-dependent adenoviral serotype 35 (Ad35) vectors. In various embodiments, helper-dependent Ad35 vectors can be used to deliver a therapeutic payload to a subject in need thereof. Exemplary payloads can encode replacement proteins, antibodies, CARs, TCRs, small RNAs, and genome editing systems. In certain embodiments, a helper-dependent Ad35 vector is engineered for integration of a payload into a host cell genome. The present disclosure further includes methods of gene therapy that include administration of a helper-dependent Ad35 vector to a subject in need thereof.

Abstract: The disclosure provides methods of diagnosing, prognosing and stratifying cancers in a patient, comprising detecting elevated expression of desmoglein-2 (DSG-2) in a sample of the cancer and referencing that level to historic data to determine treatment regimen, stage, and prognosis of a patient.

Abstract: The present invention provides recombinant adenoviral compositions and methods for their use in treating disorders associated with epithelial tissues.

Abstract: The present invention provides recombinant adenoviral compositions and methods for their use in treating disorders associated with epithelial tissues.

Abstract: The present invention provides recombinant adenoviral compositions and methods for their use in treating disorders associated with epithelial tissues.

Abstract: The present invention provides recombinant adenoviral compositions and methods for their use in treating disorders associated with epithelial tissues.

Abstract: The present invention provides recombinant adenoviral compositions and methods for their use in treating disorders associated with epithelial tissues.

Abstract: The present invention provides recombinant adenoviral compositions and methods for their use in treating disorders associated with epithelial tissues.

Abstract: The present invention provides recombinant adenoviral compositions and methods for their use in treating disorders associated with epithelial tissues.

Abstract: The present invention provides recombinant adenoviral compositions and methods for their use in treating disorders associated with epithelial tissues.

Abstract: The present invention provides recombinant nucleic acid expression cassetie and helper dependent adenovirus, where the expression cassettes utilize a miRNA based system for controlling expression of nucleases in helper dependent adenoviral viral producer cells, thus permitting production and use for in in vivo gene editing in CD34+ cells.

Abstract: The present invention provides recombinant adenoviral compositions and methods for their use in treating disorders associated with epithelial tissues.

Abstract: The present invention provides compositions, compounds, and methods relating to recombinant adenoviral-based polypeptides for treating disorders associated with epithelial tissues.

Abstract: The present invention provides recombinant adenoviral compositions and methods for their use in treating disorders associated with epithelial tissues.