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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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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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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.
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Nano-micelles used in CRISPR genome editing of mouse brains
Researchers have reported that nano-micelles can be used to efficiently deliver CRISPR-Cas9 to edit genes in the brains of mice.
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CRISPR-dead Cas9 could offer non-addictive treatment for pain, study shows
A new CRISPR gene therapy for chronic pain has been shown in mice to temporarily repress a gene involved in sensing pain.
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Machine learning used to generate AAV capsids for gene therapy
A study has used artificial intelligence to reveal adeno-associated virus (AAV) capsid variants for use in gene therapies.
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TALEN is a more efficient gene editing tool for tightly packed DNA, finds study
TALEN was shown to be almost five times more efficient than CRISPR-Cas9 at locating and editing genes in heterochromatin.
