FLORENCE, 3 July (APM) - Biotech Genespire and its Milan-based research partner San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) have developed an improved targeted gene replacement technology in human haematopoietic stem cells (HSCs), La Repubblica reported on Tuesday.
Data from a study by SR-Tiget has been published in Nature Biotechnology.
The technology is used to correct the DNA of blood stem cells through genetic editing, overcoming barriers which are typical of current techniques, the paper said. The approach opens the way to possible treatments of many pathologies such as primary immunodeficiencies, it added.
The scientists led by Luigi Naldini, director of SR-Tiget, have found one of the keys to overcoming the negative impact on haematopoietic stem cells is through one of the most important proteins for the regulation of cell proliferation, the paper explained.
The protein, know as the 'guardian of the genome', acts as a cell growth inhibitor in pathological conditions, so much so that its malfunction is associated with numerous tumours. By administering an unprecedented protein cocktail to blood stem cells during genetic editing, the researchers managed to temporarily block its action and significantly improve the efficiency of the corrective process.
It was described as an important result in the path towards clinical application in diseases in which traditional gene therapy is not indicated because of the nature of the gene to be corrected. In these cases, genetic editing offers the possibility of correcting the gene in the its natural location, maintaining its physiological regulation. It is a type of precision medicine which allows for the design of tailored therapeutic strategies, Naldini said.
The study was funded by the Telethon Foundation, the European Union's Horizon 2020 programme, the Italian health and scientific research ministries, as well as the Louis-Jeantet Foundation of Geneva.
The application of the technology to the treatment of some genetic diseases will be carried out as part of a strategic alliance between SR-Tiget and Genespire, La Repubblica said.
Hyperimmune plasma, monoclonal antibodies likely for Covid-19 therapies
Hyperimmune plasma from convalescent patients and monoclonal antibodies are the most immediate solutions for long-term therapy against Covid-19, a scientist has told parliament, Il Sole 24 Ore reported on Wednesday.
Guido Silvestri, head of the pathology department at Emory University in Atlanta and director of the Division of Microbiology and Immunology at the Yerkes National Primate Research Center, was speaking to the social affairs committee in the Chamber of Deputies.
He warned that it is still not clear how long hyperimmune plasma remains effective. But the scientist was much more positive about monoclonal antibodies.
He described them as effective treatments and noted they can be used in prevention in at-risk groups. He advised Italy to equip itself with large quantities in case of a big increase in infections further ahead.
Clinical data on drugs made with monoclonal antibodies should be available by the end of the year, which is likely to be before the availability of a vaccine, Silvestri told the committee. He cited antivirals that directly affect the virus, including remdesivir, anti-inflammatories and convalescent plasma, as other possible treatments of Covid-19.
Tuscany to allow pharmacological abortion in public health centres
Tuscany will soon allow pharmacological abortions in public health centres, according to Monday's Il Sole 24 Ore.
It will be the first region to provide for the administration of Ru486 outside hospital, although health centres will have to be linked to a hospital, regional governor Enrico Rossi told the paper.
"It is an important step forward in the development of an appropriate health service, in line with our historical approach. We were the first to buy the Ru486 abroad, considering it safer than surgical abortion," Rossi said.
The policy contrasts with neighbouring Umbria where the regional authorities recently limited pharmacological abortion by requiring all women using it to stay in hospital for three days.
Rossi said Tuscany's decision was taken some time ago. "It is completely useless to make women suffer more than they already have to. Complicating and further bureaucratising this step would only serve to blame and punish them," he said.
Initiative to involve patients in clinical trials from the start
An initiative has been launched to involve patients in clinical trials from the start, according to Wednesday's La Repubblica.
A platform for participants in studies, called 'People not just Patients", has been set up with the support of the Roche Foundation, the paper said. It will provide the means for a dialogue with patients throughout the process.
It is partly as a result of the coronavirus emergency during which trials of experimental treatments were started with the sole purpose of responding to patients' needs, the paper said.