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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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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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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.
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New gene therapy could preserve vision in retinitis pigmentosa patients
Delivering Txnip to mice with retinitis pigmentosa was an effective treatment, making this approach a potential gene therapy for the disease.
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How RNA therapeutics can be used to treat retinitis pigmentosa
Sahm Nasseri discusses promising pre-clinical results of an RNA-based therapeutic developed to treat retinitis pigmentosa type 11.
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Prime editing demonstrates success at precise gene editing in mice
Researchers have shown that prime editing is able to effectively edit genes in mice but without the off-target effects of CRISPR.
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‘Zinc finger’ gene therapy could help to treat Alzheimer’s disease
Promising results have been shown in an animal model of Alzheimer's disease treated with zinc finger protein transcription factors.
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A strategy to cloak gene therapy AAV vectors from immune responses
Dr Ying Kai Chan discusses his latest research into how the delivery of AAV vectors for gene therapies can be made safer and more effective.