Results 31 to 40 of about 2,159 (173)

Current State of Human Gene Therapy: Approved Products and Vectors

Pharmaceuticals, 2023
In the realm of gene therapy, a pivotal moment arrived with Paul Berg’s groundbreaking identification of the first recombinant DNA in 1972. This achievement set the stage for future breakthroughs.
Aladdin Y. Shchaslyvyi, Svitlana V. Antonenko, Maksym G. Tesliuk, Gennadiy D. Telegeev   +3 more
doaj   +1 more source

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, Xuefeng Li, Xiaoli Wei, Jinduan Lin, Li Ou, Li Ou   +5 more
doaj   +1 more source

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

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, Kiviniemi, Vesa, Härkönen, Ulla   +2 more
core  

Intravenous immunoglobulin prevents peripheral liver transduction of intrathecally delivered AAV vectors

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, Christian J. Hinderer, Jenny A. Greig, Cecilia Dyer, Elizabeth L. Buza, Peter Bell, Jessica A. Chichester, Peter M. Hayashi, Hanying Yan, Tamara Goode, James M. Wilson   +10 more
doaj   +1 more source

Treatment of spinal muscular atrophy with Onasemnogene Abeparvovec in Switzerland: a prospective observational case series study

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, Oswald Hasselmann, Anne Tscherter, Elea Galiart, David Jacquier, Andrea Klein   +5 more
doaj   +1 more source

Newborn screening program and advanced therapies as a chance for the youngest patients – based on spinal muscular atrophy (SMA)

Pielęgniarstwo XXI Wieku, 2023
NEWBORN SCREENING PROGRAM AND ADVANCED THERAPIES AS A CHANCE FOR THE YOUNGEST PATIENTS – BASED ON SPINAL MUSCULAR ATROPHY (SMA) Introduction. Newborn screening programs, commonly conducted, are prophylactic procedures with the aim to detect serious ...
Martyna Agnieszka Śliwińska, Anna Rakuś-Kwiatosz   +1 more
doaj   +1 more source

Overcoming the Challenges Imposed by Humoral Immunity to AAV Vectors to Achieve Safe and Efficient Gene Transfer in Seropositive Patients

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, David-Alexandre Gross, Novella Tedesco, Novella Tedesco, Christian Leborgne, Christian Leborgne, Giuseppe Ronzitti, Giuseppe Ronzitti   +7 more
doaj   +1 more source

Aspects réglementaires de la mise sur le marché d’un médicament pédiatrique innovant : étude de cas du ZOLGENSMA®

, 2023
L’Amyotrophie Musculaire Spinale (SMA) est une pathologie neuromusculaire rare d’origine génétique caractérisée par une hypotonie, une atrophie et une faiblesse musculaire progressive.
Blaisot, Élody
core  

Spinal muscular atrophy as a blueprint for precision therapy in neuromuscular disease

Expert Reviews in Molecular Medicine
Background Spinal Muscular Atrophy (SMA) is caused by a deficiency of the survival motor neuron (SMN) protein due to loss of SMN1 and inefficient compensation by SMN2. This genetic architecture has driven the development of precision therapeutics.
Busra Cetin, Melike Aliciaslan, Ezgi Erbasan, Busra Cesur Ergun, Salih Sanlioglu   +4 more
doaj   +1 more source