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New gene therapy could treat dominant optic atrophy
Researchers have found that an OPA1-targeted gene therapy can treat dominant optic atrophy in pre-clinical trials.
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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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CRISPR: a molecular scissor set to change the world
In October this year, Emmanuelle Charpentier and Jennifer Doudna, the two scientists who pioneered the revolutionary gene-editing technology CRISPR, were awarded the Nobel Prize in Chemistry. Here, Pushpanathan Muthuirulan discusses the potential for this technology and the importance of using it safely, ethically and responsibly.
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‘Neuroregenerative’ study uses glial cells for stroke repair in primates
In rhesus macaques, a team were able to induce neural regeneration from brain internal glial cells, repairing damage from stroke.
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Expert view: ddPCR whole cell DNA workflow efficiently measures success of CAR T-cell production
Not every step of CAR T-cell manufacturing can be tightly regulated, introducing some significant quality control challenges for companies developing CAR T cancer therapies
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Defusing antibody neutralisers in AAV gene therapy
Adeno-associated virus (AAV)-mediated gene therapies allow for the treatment of a growing number of diseases; however, the presence of neutralising antibodies can lead to limitations of this technology, particularly for patients who may be excluded due to these pre-existing or developing neutralising antibodies. Recently, a study was published in Nature…
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Antibodies In-Depth Focus 2020
Included in this in-depth focus are articles on antibody therapeutics to treat COVID-19, how AAV antibodies can be neutralised, the role of antibodies in cancer therapy and bispecific antibodies for immuno-oncology drugs.
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Drug Target Review – Issue 3 2020
This issue includes articles that explore how a next-generation genomics platform can be used for COVID-19 research, the elimination of neutralising AAV antibodies for gene therapies and a new quick and cost-effective biomarker technology for cancer diagnostics. Also in this issue are features on antibody therapeutics for COVID-19 and targets…
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New CRISPR system edits body’s response to prevent immunity against AAVs
A novel CRISPR system that suppresses genes related to adeno-associated virus (AAV) antibody production has been developed to prevent immunity against the gene therapy.
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Developing a nanoparticle-delivered gene therapy for blinding eye disease
Ophthalmology and engineering combine in a novel nanoparticle-delivered gene therapy approach to treating wet age-related macular degeneration.
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A new vision: restoring eyesight using a novel gene therapy technique
Scientists created a technique to give photoreceptors infrared-light sensitivity, allowing the animal models to see infrared heat signatures.
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Cancer mutation could be used to target CRISPR therapy
Researchers reveal protospacer adjacent motif mutations (PAM sites) on the NRF2 gene of cancers could be used to guide CRISPR gene editing.
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Novel gene promoters could improve gene therapies for neurological diseases
Novel promoters based on those in the herpes viruses, enabled delivery of larger genes and increased the period they were active in the nervous system.
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High-throughput screening used to analyse AAV vector libraries
Researchers have used high-throughput screening on AAV vector capsid libraries to identify which ones are best for certain gene therapies.
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Gene therapy could cure Danon disease, life-threatening genetic condition
Researchers have shown a gene therapy to correct the genetic mutation that causes Danon disease is successful in pre-clinical trials.
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TAZ gene therapy reverses Barth syndrome in mice
Researchers have successfully treated cardiac dysfuntion in mice models of Barth syndrome by using a gene therapy to replace TAZ.
