news
New angle of attack on spinal muscular atrophy (SMA)
Spinal muscular atrophy (SMA) in its most severe form is incurable and fatal in early childhood, but now researchers are mounting a multi-pronged attack for patients and their families.
List view / Grid view
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
DNA: discovering a new mechanism of epigenetic inheritance
Scientists have demonstrated the existence of transgenerational epigenetic inheritance (TEI) among Drosophila fruit flies...
news
Removing old cells could extend joint health & stop osteoarthritis
In a preclinical study in mice and human cells, researchers report that selectively removing old or 'senescent' cells from joints could stop and even reverse the progression of osteoarthritis.
news
New potential treatment for aggressive brain cancer in children
Using state-of-the-art gene editing technology, scientists have discovered a promising target to treat atypical teratoid/rhabdoid tumour (AT/RT) - a highly aggressive and therapy resistant brain tumour that mostly occurs in infants.
news
Premature cell differentiation leads to disorders in pancreatic development
Neonatal diabetes mellitus (NDM), or diabetes among infants less than six months of age, is a rare form of diabetes caused by a mutation in genes crusial to the development or function of beta cells. In about half of such cases, the disease becomes permanent (PNDM). Mutations in more than…
news
Novel genes identified that help suppress prostate and other cancers
New genes which help prevent prostate, skin and breast cancer development in mice have been discovered by researchers at the Wellcome Trust Sanger Institute and their collaborators.
news
Immunotherapy and gene therapy combination shows promise against brain tumours
The outlook for brain tumour patients is pretty negative, with the median survival rate for patients with glioblastoma multiforme, or GBM, a mere 14.2 months.
news
UK biotech Orchard Therapeutics opens US operations in California
6 October 2016 | By Niamh Louise Marriott, Digital Content Producer
Foster City will serve as headquarters for Orchard Therapeutics North America, and will provide the central base for laboratory operations associated...
news
Attendee list released for the Orphan Drugs & Rare Diseases 2016
13 September 2016 | By SMi
SMi Reports (13.09. 2016, London, UK): Alexion, BioMarin, Chiesi, MHRA, NICE to meet in London in October to discuss updates in the orphan drug landscape...
article
How NIH’s $30 million research fund will help find HIV cure
20 July 2016 | By Niamh Louise Marriott, Digital Content Producer
The NIH recently awarded $30 million to advance science towards a cure for HIV. Drug Target Review caught up with the Wistar Institute‘s Dr. Luis Montaner, co-leader of the BEAT-HIV project...
news
Mkx gene revealed as crucial for keeping tendons healthy
14 July 2016 | By Victoria White, Digital Content Producer
The Scripps Research Institute scientists behind the study say their findings may bring doctors closer to using gene therapies to grow and repair tendons...
news
AGTC gets orphan designation in the EC for XLRP gene therapy
8 June 2016 | By Victoria White, Digital Content Producer
The orphan medicinal product designation was granted to AGTC's investigational gene therapy for the treatment XLRP caused by mutations in the RPGR gene...
news
Biotechnology company Orchard Therapeutics officially launches
4 May 2016 | By Victoria White, Digital Content Producer
Biotechnology company Orchard Therapeutics has officially launched with a £21-million Series A financing led by F-Prime Capital...
news
Cell Therapy Catapult becomes Cell and Gene Therapy Catapult
2 February 2016 | By Victoria White
The Cell Therapy Catapult has changed its name to the Cell and Gene Therapy Catapult to more accurately reflect the activities of the organisation...
news
Astellas and CLINO team up for retinitis pigmentosa gene therapy
1 February 2016 | By Victoria White
Astellas and CLINO have entered into a license agreement for the worldwide development and commercialisation of Adeno-associated Virus-modified Volvox channelrhodopsin-1 (AAV-mVChR1)...
