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Use of Replicators in Gene Therapy
Abstract: Gene therapies offer promising prospects of treating a wide variety of human diseases. In one method, a gene therapy vector can be utilized to deliver an unmutated copy of a gene, called a
transgene
, to replace a mutated gene in order to treat the genetic disorder. However, lack of expression of a therapeutic transgene and uncontrolled gene...
Published: 4/8/2024
|
Inventor(s):
Mirit Aladjem
,
Cindy Tseng
,
Haiquing Fu
,
Lixin Wang
Keywords(s):
Aladjem
,
delivery system
,
DNA replication initiation
,
GENE EXPRESSION
,
Gene Silencing Modulation
,
GENE THERAPY
,
Replicators
,
TRANSGENE
Category(s):
Collaboration Sought > Licensing
,
Application > Research Materials
,
TherapeuticArea > Oncology
,
Collaboration Sought > Collaboration
Novel Codon-Optimized Gene Therapeutic for Methylmalonic Acidemia
Methylmalonic Acidemia (MMA) is a metabolic disorder characterized by increased acidity in the blood and tissues due to toxic accumulation of protein and fat by-products resulting in seizures, strokes, and chronic kidney failure. A significant portion of MMA cases stem from a deficiency in a key mitochondrial enzyme, methylmalonyl-CoA mutase (MUT),...
Published: 7/25/2024
|
Inventor(s):
Randy Chandler
,
Charles Venditti
Keywords(s):
Acidemia
,
Class
,
GB2AXX
,
GBXXXX
,
GXXXXX
,
Methylmalonic
,
Methylmalonyl-CoA
,
MMA
,
MUT
,
Mutase
,
Synthetic
,
TRANSGENE
,
treatment
Category(s):
Collaboration Sought > Collaboration
,
Application > Therapeutics
Wild-Type and DNA Polymerase beta Null Mouse Embryotic Fibroblast Cell Lines Harboring a lambda-LIZ
Transgene
Of great utility in toxicology and DNA repair research are knockout mice with cell lines enabling one to evaluate generations of gene mutations as a direct function of base excision repair. Of particular importance are lambda-LIZ
transgene
s. Likewise, wild-type and beta-pol null cell lines are equally important. While there exist cell lines carrying...
Published: 7/25/2024
|
Inventor(s):
Robert Sobol
Keywords(s):
3-@hydroxyacyl-coa dehydrogenase deficiency
,
47 XYY syndrome
,
AC5XXX
,
ACXXXX
,
AXXXXX
,
B
,
Cell
,
DNA
,
Double Y
,
Embryonic
,
Fibroblast
,
HAD deficiency
,
harboring
,
HIS deficiency
,
Histidinemia
,
KO
,
Lines
,
Mouse
,
NULL
,
polymerase
,
SV40T-Ag
,
TRANSFORMED
,
TRANSGENE
,
WILD-TYPE
,
Y-LIZ
Category(s):
Collaboration Sought > Licensing
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