FIELD: biotechnology.
SUBSTANCE: invention relates to biotechnology and genetic engineering, in particular, to nucleic acids coding acid alpha-glucosidase (GAA). Present invention also relates to expression cassettes, vectors, cells and cell lines and methods of using such nucleic acids coding acid alpha-glucosidase (GAA).
EFFECT: invention enables to use the invention as a gene therapy of individuals with Pompe disease and other diseases and disorders which can be treated using GAA.
86 cl, 21 dwg, 3 tbl, 14 ex
Title |
Year |
Author |
Number |
CPG REDUCED FACTOR VIII VARIANTS, COMPOSITIONS AND METHODS AND USE FOR TREATMENT OF HEMOSTASIS DISORDERS |
2016 |
- Anguela, Xavier
- Shen, Sam Hsien-I
|
RU2745506C2 |
MODIFIED FACTOR IX, AS WELL AS COMPOSITIONS, METHODS AND OPTIONS FOR THE USE OF GENE TRANSFER INTO CELLS, ORGANS AND TISSUE |
2016 |
- Khaj Ketrin A.
- Angela Khaver
|
RU2811445C2 |
VARIANTS OF ACID ALPHA-GLUCOSIDASE AND THEIR USE |
2017 |
- Mingozzi, Federico
- Ronzitti, Giuseppe
|
RU2780410C2 |
METHODS OF GENOTHERAPY USING FACTOR VIII (FVIII) GENE |
2018 |
|
RU2808274C2 |
OPTIONS OF ACID ALPHA-GLUCOSIDASE AND THEIR USE |
2017 |
- Mingozzi, Federico
- Ronzitti, Giuseppe
- Colella, Pasqualina
- Puzzo, Francesco
|
RU2780329C2 |
GENE THERAPY OF HYPOPHOSPHATEMIC DISEASES ASSOCIATED WITH FIBROBLAST GROWTH FACTOR 23 |
2020 |
- Rontsitti, Dzhuzeppe
- Zhoz, Luiza
- Sharl, Severin
- Mingotstsi, Frederiko
|
RU2815545C2 |
NUCLEIC ACID CONSTRUCTIONS AND VECTORS FOR GENOTHERAPY FOR TREATING WILSON'S DISEASE AND OTHER CONDITIONS |
2015 |
- Murillo Sauca, Oihana
- Gonzalez Aseguinolaza, Gloria
- Hernandez Alcoceba, Ruben
|
RU2745567C2 |
COMPOSITIONS AND METHODS FOR INTERNALIZATION OF ENZYMES |
2018 |
- Bejk, Endryu
- Signar, Katrin
- Shonkherr, Kristofer
- Kiratsus, Kristos
- Vang, Cheng
|
RU2806021C2 |
COMPOSITIONS AND METHODS FOR INCREASING OR ENHANCING TRANSDUCTION OF GENE THERAPY VECTORS AND FOR REMOVING OR REDUCING IMMUNOGLOBULINS |
2021 |
|
RU2841237C1 |
RECOVERED NUCLEIC ACID WHICH CODES FVIII-BDD-BASED FUSION PROTEIN AND HETEROLOGOUS SIGNAL PEPTIDE, AND USE THEREOF |
2022 |
- Perepelkina Mariya Pavlovna
- Vlasova Elena Veniaminovna
- Fomina Anastasiia Vladimirovna
- Gershovich Pavel Mikhailovich
- Markova Vitaliia Aleksandrovna
- Morozov Dmitry Valentinovich
|
RU2818229C2 |