CRISPR-Cas9 In Vivo Gene Editing of KLKB1 for Hereditary Angioedema
Abstract BACKGROUND Hereditary angioedema is a rare genetic disease that leads to severe and unpredictable swelling attacks. NTLA-2002 is an in vivo gene-editing therapy based
Abstract BACKGROUND Hereditary angioedema is a rare genetic disease that leads to severe and unpredictable swelling attacks. NTLA-2002 is an in vivo gene-editing therapy based
Abstract Mutations in SNCA, the gene encoding α-synuclein (αSyn), cause familial Parkinson’s disease (PD) and aberrant αSyn is a key pathological hallmark of idiopathic PD.
Highlights Summary Parkinson’s disease (PD) is a debilitating neurodegenerative disorder. Its symptoms are typically treated with levodopa or dopamine receptor agonists, but its action lacks
Abstract In myotonic dystrophy type 1 (DM1), deregulated alternative splicing of the muscle chloride channel Clcn1 causes myotonia, a delayed relaxation of muscles due to
Highlights •NPHP5-LCA patient-derived fibroblasts and RPE display abnormally elongated cilia •Outer segment protein localization is impaired in patient-derived photoreceptors •CEP290 protein reduction is observed across
Abstract Genetic intervention is increasingly being explored as a therapeutic option for debilitating disorders of the central nervous system. The safety and efficacy of gene
Hitching a ride with a retroelement Retroviruses and retroelements have inserted their genetic code into mammalian genomes throughout evolution. Although many of these integrated virus-like
Highlights •Excitotoxic and axonal injuries lead to loss of CaMKII activity in RGCs •Reactivation of CaMKII protects RGCs in multiple injury/disease models •CREB acts downstream
Abstract Uterine sensitization–associated gene-1 (USAG-1) deficiency leads to enhanced bone morphogenetic protein (BMP) signaling, leading to supernumerary teeth formation. Furthermore, antibodies interfering with binding of USAG-1
Abstract RNAi therapy has undergone two stages of development, direct injection of synthetic siRNAs and delivery with artificial vehicles or conjugated ligands; both have not
Abstract Leber congenital amaurosis due to CEP290 ciliopathy is being explored by treatment with the antisense oligonucleotide (AON) sepofarsen. One patient who was part of a larger
Abstract Developing safe and efficient non-viral delivery systems remains a major challenge for in vivo applications of gene therapy, especially in cystic fibrosis. Unlike conventional
Abstract Genetic variants account for approximately half the cases of congenital and early‐onset deafness. Methods and technologies for viral delivery of genes into the inner
Unexpected transfer Gene therapy using adeno-associated viral (AAV) vectors is a promising strategy for monogenic blinding diseases. Leber hereditary optic neuropathy (LHON) is caused by
Researchers used gene therapy to regenerate damaged axons in the eye, in a discovery that could aid the development of new treatments for glaucoma, one
Madison researchers have developed a safer and more efficient way to deliver a promising new method for treating cancer and liver disorders and for vaccination
Amyotrophic lateral sclerosis (ALS) is a debilitating and fatal disorder that can be caused by mutations in the superoxide dismutase 1 (SOD1) gene. Although ALS
Earlier this year, the US Food and Drug Administration approved the most expensive drug ever to hit the market, a gene therapy for spinal muscular
Grajevis Bakatunkanda’s mother knew the signs: when her son lost interest in dinner, that meant the pain was on its way. It would strike, like
A clinical trial of a gene therapy for Duchenne muscular dystrophy has been halted after a patient suffered serious side effects following treatment, Reuters reports
In late 2017, scientists first began attempting to edit the genes of adults to treat rare genetic disorders. Preliminary results from a clinical trial, shared
Gene therapy generally relies on delivering DNA into cells along with strategies to control its expression. Synthetic messenger RNA (mRNA) is an attractive alternative gene
A therapy that edits genes directly in the human body might be safe, suggest early findings from the first trial to test the approach. Researchers
Two studies published yesterday (October 9) in Nature Medicine report success using modified CRISPR-Cas9 gene editing to prevent or cure two inherited diseases in mice.
Researchers hope to develop treatments for a wide range of genetic disorders, and even cancer, using CRISPR-Cas9 gene editing. These clinical interventions may take the
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