CODON-OPTIMIZED NUCLEIC ACID ENCODING SMN1 PROTEIN

• Main Authors: VESELOVA, ANNA SERGEEVNA, GERSHOVICH, PAVEL MIKHAILOVICH, MOROZOV, DMITRY VALENTINOVICH, SHKLIAEVA, MARGARITA ALEKSANDROVNA, MADERA, DMITRIY ALEKSANDROVICH, SHUGAEVA, TATIANA EVGENIEVNA, LOMUNOVA, MARIA ANDREEVNA
• Format: Patent
• Language: English; French
• Published: 09.12.2021
• Subjects: BEER; BIOCHEMISTRY; CHEMISTRY; COMPOSITIONS THEREOF; CULTURE MEDIA; ENZYMOLOGY; HUMAN NECESSITIES; HYGIENE; MEDICAL OR VETERINARY SCIENCE; METALLURGY; MICROBIOLOGY; MICROORGANISMS OR ENZYMES; MUTATION OR GENETIC ENGINEERING; ORGANIC CHEMISTRY; PEPTIDES; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS; SPIRITS; VINEGAR; WINE

The present application relates to the fields of genetics, gene therapy, and molecular biology. More specifically, the present invention relates to an isolated codon-optimized nucleic acid that encodes the SMN1 protein (survival motor neuron protein), an expression cassette and a vector based thereo...