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TREM2 regulation to prevent neurodegeneration is possible, study shows
Scientists used a synthetic thyroid hormone in mice to regulate the TREM2 gene implicated in diseases such as Alzheimer’s and Parkinson’s.
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Gene Therapy
Gene Therapy is the introduction of normal genes into cells in place of missing or defective ones in order to correct genetic disorders.
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Prime editing method corrects cystic fibrosis in stem cells
A team of scientists used a CRISPR-Cas9 technique known as prime editing to correct cystic fibrosis in cultured human stem cells.
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CRISPR-Cas9 used to prevent Fuchs’ corneal dystrophy in mice
Scientists have shown that start codon disruption with CRISPR-Cas9 gene editing can prevent Fuchs’ corneal dystrophy in mouse models.
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Mitochondria repair in mice may provide answers on Parkinson’s disease
Researchers used an experimental small molecule that helped restore the removal of damaged mitochondria from brain cells in a mouse model of Parkinson's.
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Genetic treatment reverses age-related memory loss in mice
Scientists have shown that manipulating the perineuronal nets (PNNs) in the brains of mice led to the reversal of age-related memory loss.
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Prenatal editing halts progression of Hurler syndrome in pre-clinical model
US researchers used an AAV9 vector to edit a single base mutation in a prenatal mouse model, halting progression of Hurler syndrome.
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How engineered RNA binding proteins delivered in vivo by gene therapy could treat myotonic dystrophy
In this article, Dr Jim Burns discusses promising pre-clinical results of how a new platform could treat the root cause of many devastating genetic diseases including myotonic dystrophy type 1.
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Novel gene therapy for heart failure moves closer to clinic
New research by US institutions in pigs shows that knocking down a particular signalling pathway after heart failure renews heart tissue.
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Novel vectors for more efficient and less risky ocular gene therapy
Inherited blindness is the leading cause of vision loss in the working populations. Recently, the first gene therapy for the treatment of retinal dystrophy caused by mutations in the RPE65 gene became available. This gene therapy is based on adeno-associated virus (AAV) vectors injected under the retina. Subretinal injection is…
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Drug Target Review Cell & Gene Therapy ebook 2021
In this ebook, discover how organoids can be used in regenerative medicine and how a novel AAV vector for gene therapy was developed.
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Drug Target Review – Issue 2 2021
This issue includes articles on novel AAV vectors to deliver ocular gene therapy, how phenotypic models of disease are being used in covalent fragment screening and the challenges and opportunities presented by automation in the life sciences. Also in this issue are features on stem cells, antibodies and hit-to-lead.
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Nanoparticle platform enables enhanced delivery of gene therapies
Scientists have created new nanoparticle-based materials that could be used to deliver gene therapies in an adaptable way.
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Challenges and opportunities in gene therapy development
Despite the promise of gene therapies, significant challenges have emerged in the field. Dr Carsten Brunn discusses the current obstacles and opportunities when developing gene therapies.
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Novel CRISPR strategy developed for Duchenne muscular dystrophy
A CRISPR gene editing technique has been developed to restore dystrophin, which is missing in many Duchenne muscular dystrophy (DMD) patients.
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Gene therapy in Alzheimer’s mouse model preserves learning and memory
A new gene therapy that introduced SynCav1 to the brains of Alzheimer's mouse models was shown to preserve neuronal and synaptic plasticity.