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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.

news

Researchers achieve DNA methylation removal in mouse models

9 November 2021 | By Anna Begley (Drug Target Review)

The study showed that DNA “de-methylation” activity can be targeted to anywhere in the DNA and may be a new therapeutic strategy.

news

Gene therapy bolsters Parkinson’s drug benefits in pre-clinical study

3 November 2021 | By Anna Begley (Drug Target Review)

The gene therapy restored the ability of neurons to convert levodopa to dopamine and may help develop therapies to slow disease progression.

Man holding paper with DNA molecule breaking apart, representing gene editing

news

Moderna and Metagenomi to develop in vivo gene therapies

3 November 2021 | By Anna Begley (Drug Target Review)

Moderna and Metagenomi have announced a collaboration to jointly create next-generation in vivo gene editing therapeutics.

podcast

Episode 3 – Cell & gene therapy with Dr Sam Wadsworth, Ultragenyx Pharmaceutical & Dr Eric Ostertag, Poseida Therapeutics

1 November 2021 | By Drug Target Review

Tune in to this podcast to learn about AAV vectors for gene therapy delivery and engineering CAR T cells against solid tumours.

news

Gene therapy shows promise for Angelman syndrome treatment

26 October 2021 | By Anna Begley (Drug Target Review)

A new gene therapy restored motor skill-learning and usual behaviours in Angelman syndrome mouse models, suggesting a novel therapy for the condition.

news

Two-week-old human embryos reveal origin of Huntington’s disease

6 October 2021 | By Anna Begley (Drug Target Review)

Scientists have discovered a signalling pathway alteration in embryos with Huntington’s disease, paving the way for ground-breaking treatments.

Woman with rheumatoid arthritis chhecking medicines

news

Experimental vaccine a major breakthrough for rheumatoid arthritis

5 October 2021 | By Anna Begley (Drug Target Review)

A protein-based vaccine has proven a promising option to prevent rheumatoid arthritis, improving bone quality in animal models.

Doctor standing behind DNA molecules, representing gene therapy

news

Gene therapy method could restore visual function after stroke

4 October 2021 | By Anna Begley (Drug Target Review)

Scientists have revealed a way to use gene therapy to turn glial brain cells into neurons, restoring vision and potentially restoring motor function.

video

Discussing next-gen AAV vectors with Dr Arun Srivastava, University of Florida

30 September 2021 | By Drug Target Review

An interview with Dr Arun Srivastava about his research into safe and effective AAV vectors that do not prompt a reaction from the immune system.

Scientists removing eggshell from zebrafish embryo

news

CRISPR-based droplet method speeds up gene therapy research

28 September 2021 | By Anna Begley (Drug Target Review)

The new CRISPR-based technology called MIC-Drop rapidly identified several genes for heart development and function in zebrafish.

CRISPR Cas9 protein and DNA molecules, 3D gene therapy rendering

article

CRISPR-Cas9-based gene dropout screens: a powerful platform for drug discovery

23 September 2021 | By Dr Pushpanathan Muthuirulan (Harvard University)

CRISPR holds great promise in advancing pharmacological research and has fuelled the rapid expansion of using gene-edited cells for drug discovery processes. CRISPR-Cas9 dropout screens have emerged as a useful tool for high-throughput large-scale loss-of-function screens, which seek to identify the relationship between genotype and phenotype. Dr Pushpanathan Muthuirulan, Research…

whitepaper

Cell & Gene Therapy Advancements Whitepaper

16 September 2021 | By Drug Target Review

Download this whitepaper to discover the latest research and developments in cell and gene therapies via exclusive articles and interviews.

news

Gene therapy for Morquio A disease successful in rat model

14 September 2021 | By Anna Begley (Drug Target Review)

A novel gene therapy has fully corrected whole-body alterations in a rat model, paving the way for Morquio A therapies.

Brain with Alzheimer's disease - gene therapies

article

Delivering gene therapies to the brain to treat Alzheimer’s

31 August 2021 | By Victoria Rees (Drug Target Review)

Dr Ronald G Crystal, Professor and Chairman of the Department of Genetic Medicine, Weill Cornell Medical College, spoke to Drug Target Review’s Victoria Rees about his research into Alzheimer’s disease and why gene therapies represent a promising area of research for neurodegenerative conditions.

news

Brain organoids mimic autism-related head size changes

30 August 2021 | By Anna Begley (Drug Target Review)

Scientists have developed brain organoids that recapitulate the head size of autism patients to study the condition as well as possible therapies.

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Gene Therapy