Results 31 to 40 of about 1,989 (180)
A versatile toolkit for overcoming AAV immunity
Frontiers in Immunology, 2022 Recombinant adeno-associated virus (AAV) is a promising delivery vehicle for in vivo gene therapy and has been widely used in >200 clinical trials globally.
Xuefeng Li +5 more
doaj +1 more source
New challenges in health technology assessment (HTA): the case of Zolgensma
Ciência & Saúde Coletiva, 2023 Abstract Rare or orphan diseases have played an important role in the pharmaceutical industry. On the other hand, the impact of new technologies derived from genomic research has been growing in this industry, with new drugs being launched on the market at unsustainable prices for health systems and patients.
openaire +2 more sources
Immunomodulation in Administration of rAAV: Preclinical and Clinical Adjuvant Pharmacotherapies
Frontiers in Immunology, 2021 Recombinant adeno-associated virus (rAAV) has attracted a significant research focus for delivering genetic therapies to target cells. This non-enveloped virus has been trialed in many clinical-stage therapeutic strategies but important obstacle in ...
Wing Sum Chu, Joanne Ng
doaj +1 more source
BMC Neurology, 2023 Background Spinal muscular atrophy (SMA) is a rare neuromuscular disorder leading to early death in the majority of affected individuals without treatment.
Georg M. Stettner +5 more
doaj +1 more source
Molecular Therapy: Methods & Clinical Development, 2022 Gene therapy using neurotropic adeno-associated virus vectors represents an emerging solution for genetic disorders affecting the central nervous system. The first approved central nervous system-targeting adeno-associated virus gene therapy, Zolgensma®,
Makoto Horiuchi +10 more
doaj +1 more source
Zolgensma-hoito (onasemnogeeniabeparvoveekki) SMA-taudin hoidossa : Uusien sairaalalääkkeiden arviointi [PDF]
, 2020 Spinaalinen lihasatrofia (SMA) on harvinainen perinnöllinen neuromuskulaarinen sairaus. SMA-tauti johtaa lihasten rappeutumiseen ja lihasheikkouteen, mikä estää motoristen toimintojen kehittymisen ja ylläpitämisen.
Hyvärinen, Antti +2 more
core
Frontiers in Immunology, 2022 One of the major goals of in vivo gene transfer is to achieve long-term expression of therapeutic transgenes in terminally differentiated cells. The extensive clinical experience and the recent approval of Luxturna® (Spark Therapeutics, now Roche) and ...
David-Alexandre Gross +7 more
doaj +1 more source
Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology, 2023 Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disorder that causes muscle atrophy and weakness. While no specific therapies existed until a few years ago, several effective disease-modifying treatments have become available in recent years.
Bitetti, Ilaria +3 more
openaire +2 more sources
Frontiers in Neurology, 2020 Spinal Muscular Atrophy (SMA) is monogenic motoneuron disease caused by low levels of the Survival of Motoneuron protein (SMN). Recently, two different drugs were approved for the treatment of the disease.
Niko Hensel +5 more
doaj +1 more source
Frontiers in Molecular Neuroscience, 2021 In recent years, gene therapy has been raising hopes toward viable treatment strategies for rare genetic diseases for which there has been almost exclusively supportive treatment. We here review this progress at the pre-clinical and clinical trial levels
Thomas Leth Jensen +2 more
doaj +1 more source