C12Y203/0105

MODULATION OF SPTLC1 VIA RECOMBINANT ADENO-ASSOCIATED VECTORS

The disclosure provides, in some aspects, compositions (e.g., isolated nucleic acids and rAAVs) comprising a transgene which encodes at least one inhibitory nucleic acid which decreases expression of a gene encoding a serine palmitoyltransferase protein (e.g., SPTLC1). In some aspects, the disclosure relates to methods of treating hereditary sensory and autonomic neuropathy 1 (HSAN1) by administering the compositions described by the disclosure to a cell or subject.

Method for producing sphingoid base or sphingolipid

A method for producing an objective substance such as sphingoid bases and sphingolipids using yeast is provided. An objective substance is produced by cultivating yeast having an ability to produce the objective substance in a culture medium containing an additive that is able to associate with, bind to, solubilize, and/or capture the objective substance, and collecting the objective substance from cells of the yeast and/or the culture medium.

ALLELE-SPECIFIC GENE SUPPRESSION
20190144869 · 2019-05-16 ·

The invention relates to an oligonucleotide that supresses the expression of an allele carrying a dominant mutation that causes hereditary sensory neuropathy type I (HSN1), wherein the suppression takes place through hybridisation of said oligonucleotide to the DNA of said allele or to an RNA transcript of said allele, and which either does not suppress the expression of a wild-type allele not containing the dominant mutation or suppresses the expression of said wild-type allele to a lesser extent than it suppresses the expression of the dominant mutant allele; and also pharmaceutical compositions comprising oligonucleotides of the invention and treatments of HSN1 using such oligonucleotides.

TARGETING 3-KETODIHYDROSPHINGOSINE REDUCTASE (KDSR) IN CANCER
20240342255 · 2024-10-17 ·

Provided herein are methods for treating cancer using an inhibitor of KDSR, a precursor of 3KDS (e.g., palmitate), 3KDS itself, or a combination thereof.

METHOD FOR PRODUCING SPHINGOID BASE OR SPHINGOLIPID

A method for producing an objective substance such as sphingoid bases and sphingolipids using yeast is provided. An objective substance is produced by cultivating yeast having an ability to produce the objective substance in a culture medium containing an additive that is able to associate with, bind to, solubilize, and/or capture the objective substance, and collecting the objective substance from cells of the yeast and/or the culture medium.

Pichia ciferrii cells and uses thereof

The invention relates to genetically modified Pichia ciferrii cells, to the use thereof and to a method of producing sphingoid bases and sphingolipids.

PREPARATION METHOD OF PECTIC POLYSACCHARIDE WITH EFFECT OF REGULATING AND CONTROLLING ICE CRYSTAL GROWTH

The present disclosure provides a preparation method of a pectic polysaccharide with an effect of regulating and controlling ice crystal growth, comprising step 1, extracting a pectin crude extract from fruit and vegetable powder; step 2, carrying out branched chain enzymolysis on pectin crude extract to obtain pectin enzymatic hydrolysate; step 3, adding pectin methylesterase and pectin acetylesterase into pectin enzymatic hydrolysate for precise de-esterification enzymolysis; step 4, adding 0.5-2 U/mL of polygalacturonase into pectin enzymatic hydrolysate obtained in step 3, and hydrolyzing for 1.5-2.5 hours; and step 5, collecting precipitate in pectin enzymatic hydrolysate obtained in step 4 to obtain modified pectin. The modified pectin has a purity higher than 90%, a methyl esterification degree of 45-55%, an acetylation degree less than 2%, a neutral sugar content less than 5%, and an average molecular weight of 1.4-7.5 kDa, and obviously reduces an ice crystal size in a freezing process.

Modulation of SPTLC1 via recombinant adeno-associated vectors

The disclosure provides, in some aspects, compositions (e.g., isolated nucleic acids and rAAVs) comprising a transgene which encodes at least one inhibitory nucleic acid which decreases expression of a gene encoding a serine palmitoyltransferase protein (e.g., SPTLC1). In some aspects, the disclosure relates to methods of treating hereditary sensory and autonomic neuropathy 1 (HSAN1) by administering the compositions described by the disclosure to a cell or subject.

MUTANT WICKERHAMMYCES CIFERRll STRAIN HAVING IMPROVED PRODUCTIVITY FOR TAPS, AND METHOD FOR PRODUCING TAPS USING SAME

The present invention relates to a Wickerhamomyces ciferrii mutant strain having an improved ability to produce tetraacetylphytosphingosine (TAPS), and a method for producing TAPS using the same. The Wickerhamomyces ciferrii mutant strain provided by the present invention exhibits excellent TAPS productivity, and thus may be widely used in development of various products that use TAPS.