Genetic/congenital - Articles - Page 27

Endocrine/Metabolic

NGGT-002 gene therapy shows potential for treating phenylketonuria

May 29, 2023

NGGT (Suzhou) Biotechnology Co. Ltd. has presented preclinical data on an AAV vector approach that expresses human PAH, rAAV8-PAH, also known as NGGT-002. NGGT-002 has liver tropism and it was codon-optimized for expressing PAH in the liver.

Neurology/Psychiatric

Researchers create new mouse model of DMD using knock-in of human exon 50

May 26, 2023

Researchers from Huidagene Therapeutics Co. Ltd. have evaluated the effects of adenine base editing (ABE)-induced exon skipping of exon 50 in a humanized mouse model of Duchenne muscular dystrophy (DMD).

Endocrine/Metabolic

AAV9-CLN5 improves symptoms in mice with Batten disease

May 26, 2023

Neuronal ceroid lipofuscinosis, commonly known as Batten disease, is an inherited pediatric neurodegenerative lysosomal storage disease caused by mutations in the CLN5 gene. The disease is incurable and there is an urgent medical need for novel therapies. A murine model of Batten disease was developed to study a novel AAV vector that expresses CLN5, AAV9-CLN5. In the study by University College London investigators, the gene therapy, driven by the synapsin promoter, was intracerebroventricularly administered into neonatal Cln5-knockout mice.

Neurology/Psychiatric

MyoDys45-55 restores dystrophin and improves functional measures in murine model of DMD

May 26, 2023

Myogene Bio LLC has developed an approach through CRISPR7Cas9 technology...

Endocrine/Metabolic

AAV9-GLA restores α-galactosidase levels in murine model of Fabry disease

May 25, 2023

Fabry disease is a metabolic disease characterized by a deficiency in the lysosomal α-galactosidase enzyme caused by mutations in the GLA gene. This leads to substrate accumulation in the lysosomes, cellular dysfunction and organ damage.

Genetic/Congenital

Poseida Therapeutics develops a new hybrid approach for OTCD treatment

May 23, 2023

Ornithine transcarbamylase deficiency (OTCD) is an X-linked genetic condition, the most common...

Genetic/Congenital

Ex vivo autologous gene therapy risk leaves room for improvement

May 22, 2023

By Mar de Miguel

Gene therapy technology makes it possible to select diseased or mutated cells from a patient, modify them in the laboratory and reintroduce them to the body to treat different disorders. This is known as ex vivo autologous gene therapy. The difference with allogeneic cell techniques is whether the donor is oneself (autologous) or a compatible person (allogeneic), which would provide healthy cells that do not need genetic modification.

The DNA double helix overlays a field of ACGTs and binary numbers.

Genetic/Congenital

Gene editing advances progress, by moving three steps forward and two steps back

May 19, 2023

By Mar de Miguel

The discovery of DNA was a milestone in the history of science that led to a breakthrough in biomedical research. By associating disease and genetics, genome correction techniques were ultimately developed that are supposed to work in the same way that antibiotics and antivirals block pathogenic microorganisms: by directly attacking the causes of disease.

Concept art for prenatal genetic testing and whole genome sequencing.

Neurology/Psychiatric

Placental gene expression sets risk trajectory for schizophrenia

May 17, 2023

By Anette Breindl

By analyzing gene expression patterns in the placenta of nearly 150 pregnancies and comparing them to fetal gene expression in the brain, researchers from the Lieber Institute for Brain Development have gained new insights into the importance of placental tissue in setting the risk trajectory for the development of schizophrenia. The work was published in Nature Communications on May 15, 2023.

Genetic/Congenital

Pangenome gives more panoramic view of human diversity

May 10, 2023

By Mar de Miguel

The human genome, the sequence that represents the DNA of our species, was built with a single individual as a model. This all-in-one standard didn’t include the gene variations that make us different or explain why some people develop certain diseases. Four simultaneous studies from the Human Pangenome Reference Consortium have published a sequence based on 47 individuals, beginning to capture the genetic diversity that defines humans.

Genetic/congenital, BioWorld Science
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NGGT-002 gene therapy shows potential for treating phenylketonuria

Researchers create new mouse model of DMD using knock-in of human exon 50

AAV9-CLN5 improves symptoms in mice with Batten disease

MyoDys45-55 restores dystrophin and improves functional measures in murine model of DMD

AAV9-GLA restores α-galactosidase levels in murine model of Fabry disease

Poseida Therapeutics develops a new hybrid approach for OTCD treatment

Ex vivo autologous gene therapy risk leaves room for improvement

Gene editing advances progress, by moving three steps forward and two steps back

Placental gene expression sets risk trajectory for schizophrenia

Pangenome gives more panoramic view of human diversity

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