A 24‑week pregnant woman fears for her unborn baby, who is developing with a sacrococcygeal teratoma so large and vascularized that it nearly surpasses the size of the fetus itself. Faced with this threat, surgeons operate inside the uterus in an open procedure that partially exposes the baby to remove the tumor and give the baby a chance to survive until birth. According to scientists presenting at the American Society of Gene & Cell Therapy's special meeting on Breakthroughs in Targeted In Vivo Gene Editing, this could be avoided.
Kedrion SpA’s investigational plasma-derived treatment for congenital aceruloplasminemia has been awarded European orphan drug designation by the EMA. The company is working to advance this treatment toward clinical development in Europe.
The number of deaths caused by prion diseases reaches about 30,000 annually. Only 5 months pass from the diagnosis of seemingly healthy patients to the fatal outcome of this neurodegenerative condition, and just 1 month until quality of life is completely lost. Removing the brain protein that causes this genetic or infectious disorder could be achieved thanks to new gene-silencing techniques. At a special meeting of the American Society of Gene & Cell Therapy, in “AAV-mediated epigenetic editing for prion disease,” Sonia Vallabh presented not just the data of her research, but the impact of this disease on her family and on herself.
The field of gene therapy is experiencing major advances driven by precise editing technologies, such as base and prime editing, and by the design of increasingly sophisticated vectors to deliver payloads that could reverse the effects of diseases. However, in the transition to in vivo applications many approaches still fail in their attempt to effectively reach target tissues or cells.
A 24‑week pregnant woman fears for her unborn baby, who is developing with a sacrococcygeal teratoma so large and vascularized that it nearly surpasses the size of the fetus itself. Faced with this threat, surgeons operate inside the uterus in an open procedure that partially exposes the baby to remove the tumor and give the baby a chance to survive until birth. According to scientists presenting at the American Society of Gene & Cell Therapy's special meeting on Breakthroughs in Targeted In Vivo Gene Editing, this could be avoided.
The field of gene therapy is experiencing major advances driven by precise editing technologies, such as base and prime editing, and by the design of increasingly sophisticated vectors to deliver payloads that could reverse the effects of diseases. However, in the transition to in vivo applications many approaches still fail in their attempt to effectively reach target tissues or cells.
A significant share of the risk and heritability of attention-deficit hyperactivity disorder (ADHD) is explained by rare genetic variants. A study led by scientists from Aarhus University in Denmark has uncovered their weight in this condition and identified three variants that will help to better understand their role, the risk of developing it, or its comorbidities, in contrast with the common and more frequent variants associated with ADHD.
Cystic fibrosis (CF) is a genetic disorder affecting around 90,000 people worldwide. It is commonly caused by the ΔF508 mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which results in a misfolded CFTR protein that is subsequently ubiquitinated and degraded.
Montse Rosa Therapeutics Inc. has developed a molecular glue degrader named MRT-2359 that selectively degrades the translation termination factor ERF3A.
Researchers from Sionna Therapeutics Inc. presented preclinical efficacy data on first-in-class NBD1 stabilizers and their use in combination with complementary modulators to correct cystic fibrosis transmembrane conductance receptor (CFTR) assembly.
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