The present disclosure relates to vectors for cloning and expressing genetic material including but not limiting to antibody gene or parts thereof and methods of generating said vectors. Said vectors express the antibody genes in different formats such as Fab or scFv as a part of intertransfer system, intratransfer system or direct cloning and expression in individual display systems. In particular, phage display technology is used to clone and screen potential antibody genes in phagemid which is followed by the transfer of said genes to yeast vector for further screening and identification of lead molecules against antigens. The present vectors have numerous advantages including uniquely designed inserts/expression cassettes resulting in efficient and smooth transfer of clonal population from phage to yeast vectors resulting in efficient library preparation and identification of lead molecules.

Embodiments of the invention are generally directed to compositions and methods of delivering one or more transgene to a target cell, such as a tumor cell, in a site-specific manner to achieve enhanced expression and to constructs and compositions useful in such applications. In certain aspects, expression from a therapeutic nucleic acid may be assessed prior to administration of a treatment or diagnostic procedure to or on a subject.

The present invention relates to viral vectors that are capable of delivering a heterologous gene to the retina and in particular delivering RLBP1 to RPE and Müller cells of the retina. The invention also relates nucleic acids useful for producing viral vectors, compositions comprising the viral vectors and uses of the compositions and viral vectors. The invention also relates to methods of delivering and/or expressing a heterologous gene to the retina, improving the rate of dark adaption in a subject and treating RLBP1-associated retinal dystrophy.

A modified Herpes Simplex Virus (HSV), which has a portion of gD (glycoprotein D) of the glycoproteic envelope deleted and a heterologous single chain antibody inserted in place of such deleted portion; the modified HSV is capable of infecting cells through receptor HER2/ErbB2 but not through receptors HVEM/HveA and nectin1/HveC; uses of the modified HSV and a process of the preparation thereof are also disclosed.

Methods, systems, compositions and strategies for the delivery of WW domain-containing fusion proteins into cells in vivo, ex vivo, or in vitro via ARMMs are provided. Methods, systems, compositions and strategies for the delivery of Cas9 proteins and/or Cas9 variants into cells in vivo, ex vivo, or in vitro via fusion to ARMM associated proteins (e.g., ARRDC1 or TSG101) are also provided.

Provided herein are genetically modified arenaviruses suitable as vaccines against mycobacterial infections. The invention also relates to pharmaceutical compositions and methods for the prevention and treatment of mycobacterial infections. Specifically, provided herein are pharmaceutical compositions, vaccines, and methods of preventing and treating infections in Mycobacterium tuberculosis.

Materials and Methods related to gene targeting (e.g., gene targeting with transcription activator-like effector nucleases; “TALENS”) are provided.

The disclosure relates to optimized polynucleotide sequences for LAMP-2B, expression cassettes, vectors, and methods of use thereof in treating disease, e.g. Danon disease.

The present disclosure provides a method for genetically engineering Yarrowia lipolytica host cell for producing glycolic acid from organic wastes. A subject genetically engineered Y. lipolytica cell comprises the disrupted native genes encoding malate synthase, heterologous enzyme of glyoxylate reductase targeted in the different cellular compartments including mitochondria, peroxisome and cytosol, and a mutant NADP.sup.+-dependent malate dehydrogenase. The pathway with a theoretical yield as high as that 1 g of acetic acid can be converted to 1.27 g of glycolic acid without carbon loss was engineered for glycolic acid production. The methods particularly include process for production of volatile fatty acids (VFAs) mainly comprised of acetic acid from organic waste, and then use of resultant VFAs for biosynthesis of glycolic acid by recombinant Y. lipolytica.

The present invention relates to viral vectors that are capable of delivering a heterologous gene to the retina and in particular delivering RLBP1 to RPE and Müller cells of the retina. The invention also relates nucleic acids useful for producing viral vectors, compositions comprising the viral vectors and uses of the compositions and viral vectors. The invention also relates to methods of delivering and/or expressing a heterologous gene to the retina, improving the rate of dark adaption in a subject and treating RLBP1-associated retinal dystrophy.