Results 21 to 30 of about 5,948,874 (305)
Gene Therapy for Regenerative Medicine
, 2023 The development of biological methods over the past decade has stimulated great interest in the possibility to regenerate human tissues. Advances in stem cell research, gene therapy, and tissue engineering have accelerated the technology in tissue and ...
Ghorbanali Sharifzadeh +3 more
core +1 more source
Durability of transgene expression after rAAV gene therapy [PDF]
, 2022 Recombinant adeno-associated virus (rAAV) gene therapy has the potential to transform the lives of patients with certain genetic disorders by increasing or restoring function to affected tissues.
Schulz, Martin +4 more
core +1 more source
Molecular Metabolism, 2017 Objective: Type 1 diabetes is characterized by autoimmune destruction of β-cells leading to severe insulin deficiency. Although many improvements have been made in recent years, exogenous insulin therapy is still imperfect; new therapeutic approaches ...
Cristina Mallol +10 more
doaj +1 more source
Proceedings of the National Academy of Sciences, 1997 In recent years, there have been a number of technological breakthroughs that have allowed for clinical trials in gene therapy to be initiated. In combination with the genome initiative, the potential for new therapeutics is limitless. Although an enormous amount of information has been obtained in a relatively short period of time, gene therapy ...
M A, Kay, D, Liu, P M, Hoogerbrugge
openaire +2 more sources
Redosing Adeno-Associated Virus Gene Therapy to the Central Nervous System
, 2022 Adeno-associated virus (AAV)-mediated gene therapies have provided promising treatments for numerous neurological disorders. Redosing of AAV to the central nervous system (CNS) is an attractive research area due to both the somewhat immunologically ...
Arjomandnejad, Motahareh +4 more
core +1 more source
Scientific Reports, 2022 As photoreceptor cells die during retinal degeneration, the surrounding microenvironment undergoes significant changes that are increasingly recognized to play a prominent role in determining the efficacy of therapeutic interventions.
A. Matsuyama +4 more
doaj +1 more source
Antibody-based CCR5 blockade protects Macaques from mucosal SHIV transmission
Nature Communications, 2021 CCR5 is a co-receptor for many transmitted HIV strains. Here, the authors show that biweekly injection of the CCR5-specific antibody Leronlimab protects rhesus macaques against infection following repeated intrarectal challenges of a CCR5-tropic SHIV.
Xiao L. Chang +32 more
doaj +1 more source
Nature Communications, 2020 Adeno-associated viruses (AAVs) are vehicles for gene therapy in humans, but currently only a limited amount of AAV serotypes is available. Here, the authors identify a novel AAV, AAVv66, and demonstrate enhanced production yields, virion stability, and ...
Hung-Lun Hsu +14 more
doaj +1 more source
Scientific Reports, 2017 The strict species specificity of Human Cytomegalovirus (HCMV) has impeded our understanding of antiviral adaptive immune responses in the context of a human immune system.
Lindsey B. Crawford +7 more
doaj +1 more source
Skeletal Muscle, 2017 Background Previous studies in patients with limb-girdle muscular dystrophy type 2A (LGMD2A) have suggested that calpain-3 (CAPN3) mutations result in aberrant regeneration in muscle.
Mehmet E. Yalvac +9 more
doaj +1 more source