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An update on gene therapy for Spinal muscular atrophy

Dr Rajvinder Karda summarises gene therapy for Spinal muscular atrophy.

First in man in vivo delivery of CRISPR-Cas9 targeting Transthyretin Amyloidosis

This year the field of genome editing has experienced unprecedented momentum.

With CRISPR basking in the limelight following on from the joint Emmanuelle Charpentier and Jennifer A. Doudna 2020 Nobel Prize there has been a phenomenal growth in R&D and industry partners creatively exploring the platform’s capabilities.

Vaccines for COVID-19

BSGCT President Elect, Prof. Rafael Yáñez-Muñoz, takes a closer look at the COVID-19 vaccines.

A Commentary on Vaccine-Induced Immune Thrombotic Thrombocytopenia

A commentary on the link between vaccination and thrombotic events (blood clots) with thrombocytopenia (low platelet count), termed vaccine-induced immune thrombotic thrombocytopenia (VITT).

See you LATER to pain

Treatment of chronic pain in an effective yet safe manner represents an unmet clinical need that requires urgent novel strategies. One such approach investigated by Ana Moreno and colleagues from the University of California, involves the use of gene therapy to dampen a specific component of the pain signalling pathway.

Addressing the challenge of informing the wider population on advances in gene and cell therapy

The role for the BSGCT in engaging with the public is becoming ever more important as an increasing number of gene and cell therapies are either licensed for clinical use or are currently in, or are reaching the stage of recruiting patients for, clinical trials.

ERC Horizon Europe 2021 Submission Dates

ERC Horizon Europe 2021 funding
calls open. View the work programme
and calendar for grant competitions.

Season's Greetings from BSGCT

View our end of year message

Precision genome editing for mitochondrial DNA – Mission accomplished?

A new study conducted by Mok et al. has described a bacterial toxin with the ability to perform precise editing in the previously ‘untouchable’ mitochondrial DNA (mtDNA) genome.

Switching Up T Cell Therapies

With the huge effort that’s currently being spent developing more resistant T cell therapies with new targets and increased efficacy against solid tumours, it’s easy to overlook the toxicity concerns and the innovative ways scientists are tackling them.

Could It Be A One-Stop Solution For The Most Common Cause Of Irreversible Blindness?

Vered Smith is our 2020 Science Writing Competition winner with her article about glaucoma.

Rebuilding the brain with stem cells: is it a no-brainer?

Anitta Rose Chacko is our 2020 Science Writing Competition runner-up with her article about Parkinson's disease.

The Churchillian dilemma of Gene Editing

Felicity Crawshay-Williams is our 2020 Science Writing Competition runner-up with her article about the genetic code.

Skin; Lets Break It Down

Christopher Smith receives a special mention for our 2020 Science Writing Competition with his poem about the SGPL1 gene.

Programmed to kill: Synthetic virology to attack cancer

Carefully modified viruses are becoming major new drugs in the battle against cancer.

Gene-loaded sutures to promote tendon healing

Injuries to tendons, the tissue that links muscle to bone, are commonly seen in the clinic yet few strategies exist to improve upon slow and largely poor tendon healing standards.

3D Printing of Personalized Cardiac Patches and Hearts

Tissue engineering for cardiac repair has reached an advanced stage and engineered constructs are starting to be approved for first-in-human trials.

Retirement of Prof George Dickson, a founder of British Society for Gene and Cell Therapy

The British Society for Gene Therapy, as BSGCT was originally named, was founded seventeen years ago by a number of pioneers in the field including Prof George Dickson.

The origin of your noggin

‘Where do you get your brains from?!’

Human Germline Editing – What’s the big deal?

In November 2018, He Jiankui, gave a presentation at the second international summit on Human Genome Editing.

One image at a time: using cryoEM to unpick how viruses infect cells

Over the last few years a wave of virus-based therapies has progressed through late stage clinical evaluation and to (or near to) the market place.

A final chance to breathe: Could gene therapy cure cystic fibrosis lung disease?

For decades, scientists and doctors have worked tirelessly towards developing effective treatments for CF.

Cracking the enthesis code

More than three quarters of shoulder repair surgeries fail while long-term disabilities occurring as a direct consequence of sports-related injuries, such as pain and osteoporosis, remain a huge burden (Apostolakos et al, 2014).

Overcoming the disconnection

We all have a universe inside our head. A hundred billion neurons making connections with one another, constantly firing and passing messages from our brain, down the spinal cord, and to every part of our body.

Putting the dopamine back in Parkinson’s

One in 37 people in the UK are diagnosed with Parkinson’s disease (PD) during their lifetime (Parkinson’s UK, 2018).

Rare is not so rare

In the disease surfactant protein B deficiency, a certain protein called surfactant protein B is missing in the lung of new-born babies.

CRISPR-engineered T cells in patients with refractory cancer

A recent paper from Carl June’s lab published in the journal Science¹ described results of a University of Pennsylvania clinical trial that administered in three patients T-cells engineered to target cancer cells using a virus whilst simultaneously engineered using CRISPR to stop expression of 3 genes.

Precision correction of pathogenic mutations using prime editing technology

A broad range of genome editing tools are currently available enabling targeted scission at a predetermined DNA locus.

It’s an ECR life?

Who'd be an Early Career Researcher these days? Long gone are the halcyon days where you complete your PhD then walk straight into a permanent academic role.

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