摘要:
Provided are gene therapy technologies, including specifically designed and tested guide RNA sequences for improved base editors, useful for increasing the expression of the gamma-globin gene. The guide RNA sequences may target the BCL11A erythroid enhancer or the gamma-globin promoter, or both at the same time. The base editors can include nucleobase deaminase inhibitor that inhibits the editing activity of the base editors until they are bound to the target sites. These gene therapy technologies are useful for treating diseases including beta-thalassemia and sickle cell anemia, among others.
摘要:
The inventors have unexpectedly discovered that shock and/or potential multi-organ failure due to shock can be effectively treated by administration of liquid high-dose protease inhibitor formulations to a location upstream of where pancreatic proteases are introduced into the gastrointestinal tract. Most preferably, administration is directly to the stomach, for example, via nasogastric tube under a protocol effective to treat shock by such administration without the need of providing significant quantities of the protease inhibitor to the jejunum and/or ileum.
摘要:
The disclosure relates to supplement formulations comprising amino acids in ratios that are protein-specific. Described herein is a protein-specific supplement formulation comprising amino acids in the ratio present in the protein's polypeptide sequence. Further described herein is a protein-specific supplement formulation comprising: a base protein and essential amino acids; wherein the relative amounts of amino acids in the formulation match the relative amounts of amino acids naturally occurring in the protein.
摘要:
The invention provides a method for the isolation of haptoglobin from a sample comprising Cohn fraction V, wherein said method comprises anion exchange chromatography of said sample. The product may be further purified and/or subjected to one or more virus inactivation or reduction steps. The isolated haptoglobin may then be formulated for pharmaceutical use.
摘要:
The present invention relates to methods and uses of cells for the prevention and treatment of a wide variety of diseases and disorders and the repair and regeneration of tissues and organs using low passage and extensively passaged in vitro cultured, self-renewing, colony forming somatic cells (CF-SC). For example, adult bone marrow-derived somatic cells (ABM-SC), or compositions produced by such cells, are useful alone or in combination with other components for treating, for example, cardiovascular, neurological, integumentary, dermatological, periodontal, and immune mediated diseases, disorders, pathologies, and injuries.
摘要:
A composition for effectively preventing or treating peptic ulcers not caused by Helicobacter pylori that does not cause any adverse reaction and thus is highly safe is provided. The present invention relates to a composition for preventing and/or treating peptic ulcers not caused by Helicobacter pylori, which comprises as an active ingredient a browning reaction product of a sugar and a protein.
摘要:
The present invention relates to methods comprising administering to an individual regulating body weight calcium-containing whey products in an amount effective to maintain muscle mass, induce weight and/or fat loss, prevent weight and/or fat gain, and/or increase the metabolic consumption of adipose tissue in the individuals.
摘要:
Provided are catechol nanoparticles, catechol protein nanoparticles, and a preparation method and use thereof. The method includes: adding a tannin compound-containing natural herb medicine into water to obtain a mixture, and subjecting the mixture to heating reflux extraction to obtain a herb medicine extract and subjecting the herb medicine extract to fractionation to obtain the catechol nanoparticles.
摘要:
Methods and compositions for promoting donor-specific tolerance and immunocompetence to a recipient of a solid organ transplant, by implanting an allogeneic solid organ in a recipient in need of a solid organ transplant and further comprising surgical implantation of a tissue-engineered allogeneic cultured postnatal thymus tissue product in the recipient of a solid organ from a donor.