One of the trending fields in almost all areas of science and technology is artificial intelligence. Computational biology and artificial intelligence can help gene therapy in many steps including: gene identification, gene editing, vector design, de...
5q-spinal muscular atrophy (SMA) is a neuromuscular disorder (NMD) that has become one of the first 5% treatable rare diseases. The efficacy of new SMA therapies is creating a dynamic SMA patient landscape, where disease progression and scoliosis dev...
Adrenoleukodystrophy is a genetic metabolic disorder characterized by a heterogeneous phenotype. Its severe form, known as cerebral adrenoleukodystrophy, involves unpredictable cerebral damage and progressive central nervous system deterioration. Thi...
The emergence of Artificial Intelligence (AI) and its usage in regenerative medicine represents a significant opportunity that holds the promise of tackling critical challenges and improving therapeutic outcomes. This article examines the ways in whi...
Here, we present a protocol for the rapid functional screening of gene editing and addition strategies in patient-derived organoids using the deep-learning-based tool DETECTOR (detection of targeted editing of cystic fibrosis transmembrane conductanc...
PURPOSE: To quantify outer retina structural changes and define novel biomarkers of inherited retinal degeneration associated with biallelic mutations in RPE65 (RPE65-IRD) in patients before and after subretinal gene augmentation therapy with voretig...
Adeno-associated virus (AAV) has emerged as a leading vector for gene therapy due to its broad host range, low pathogenicity, and ability to facilitate long-term gene expression. However, AAV vectors face limitations, including immunogenicity and ins...
The convergence of artificial intelligence (AI) and nanomedicine has revolutionized the design of smart multifunctional nanocarriers (SMNs) for drug and gene delivery, offering unprecedented precision, efficiency, and personalization in therapeutic a...
Adeno-associated virus (AAV)-based therapeutics have the potential to transform the lives of patients by delivering one-time treatments for a variety of diseases. However, a critical challenge to their widespread adoption and distribution is the high...
Molecular therapy : the journal of the American Society of Gene Therapy
40176349
Adeno-associated virus (AAV) has emerged as a highly promising vector for human gene therapy due to its favorable safety profile, versatility, and ability to transduce a wide range of tissues. However, natural AAV serotypes have shortcomings, includi...