Nucleases and methods of using these nucleases for inserting a sequence encoding a therapeutic protein such as an enzyme into a cell, thereby providing proteins or cell therapeutics for the provision of proteins lacking or deficient in subjects with a lysosomal storage disease and treatment and/or prevention of lysosomal storage diseases.

Nucleases and methods of using these nucleases for inserting a sequence encoding a therapeutic protein such as an enzyme into a cell, thereby providing proteins or cell therapeutics for the provision of proteins lacking or deficient in subjects with a lysosomal storage disease and treatment and/or prevention of lysosomal storage diseases.

Nutritional formulas comprising long-chain polyunsaturated fatty acids (LC-PUFAs) are provided, along with methods and devices for preparing and/or administering nutritional formulas. In some embodiments, a percentage of the LC-PUFAs in the nutritional formula are in the form of monoglycerides and/or free fatty acids. In some embodiments, the nutritional formulas do not comprise added lipase. Also provided are methods for providing nutrition to a subject, methods for improving fat absorption, methods for improving cognitive ability, methods for preventing chronic lung disease, and methods for reducing the length of time a patient requires total parenteral nutrition.

The present application provides a method for preparing a medical product for covering tissue, the method comprising providing nanofibrillar cellulose, providing a bioactive molecule, and covalently bonding the bioactive molecule to the nanofibrillar cellulose. The present application also provides a medical product for covering tissue comprising a bioactive molecule covalently bound to nanofibrillar cellulose.

The present application provides a method for preparing a medical product for covering tissue, the method comprising providing nanofibrillar cellulose, providing a bioactive molecule, and covalently bonding the bioactive molecule to the nanofibrillar cellulose. The present application also provides a medical product for covering tissue comprising a bioactive molecule covalently bound to nanofibrillar cellulose.

The present invention features compositions and methods for editing deleterious mutations associated with hemoglobinopathies, such as sickle cell disease (SCD). In particular embodiments, the invention provides methods for correcting mutations in a beta globin polynucleotide using modified adenosine base editors termed “ABE8” having unprecedented levels (e.g., >60-70%) of efficiency.

The disclosure provides adenosine deaminases that are capable of deaminating adenosine in DNA to treat cancers, such as melanoma and glioblastoma. The disclosure also provides fusion proteins, guide RNAs and compositions comprising a Cas9 (e.g., a Cas9 nickase) domain and adenosine deaminases that deaminate adenosine in DNA, for example in a STAT3 gene. In some embodiments, adenosine deaminases provided herein are used to modify the STAT3 gene so that its protein product, STAT3, is unable to be activated. In some embodiments, the methods and compositions provided herein are used to treat melanoma or glioblastoma.

A method for the prevention, treatment, or amelioration of a medical disease or condition associated with inflammation caused by glutamate excitotoxicity comprising administering to a patient a peptide that binds to or interferes with the P38 MAPK-COX2-PGE.sub.2 and ERK MAPK-CX3CL1-sCX3CL1 pathways.

A probiotic composition comprised of a probiotic, a prebiotic, a complex-starch reducing enzyme, an enzymatic co-factor, and a pH modifier. The probiotic composition facilitates the creation of a biofilm within the GI tract of poultry that outcompetes and excludes pathogens. The probiotic composition can be incorporated into an eggwash and sprayed onto an egg, the sprayed egg developing a stable probiotic population within the GI tract of the pre-hatched chick. The probiotic composition can also be incorporated into poultry feed by applying the probiotic composition as a mash prior to pelleting. The probiotic composition can also be applied post-pelleting as a wet or dry spray, directly onto the pellets. In some instances, the probiotic composition can be added a drinking water source used by poultry as a water additive. Poultry that consume feed or water that contain the probiotic composition develop a stable probiotic population within their GI tracts.

Compositions and methods for treating cancer that include administering a therapeutically effective amount of a tumor suppressor mRNA complexed with a delivery vehicle as described herein, e.g., a nanoparticle.