Ex vivogene modification therapy for genetic skin diseases—recent advances in gene modification technologies and delivery
نویسندگان
چکیده
منابع مشابه
Modification of Baculovirus for Gene Therapy
Gene therapy has the potential to treat devastating inherited diseases for which there is little hope of finding a conventional cure and is a method for the prevention, correction, or modulation of genetic and acquired diseases that uses genes to therapeutic proteins. Animal viral systems are by far the most effective means of gene delivery and achieve high efficiencies for both delivery and ex...
متن کاملRecent Advances in Hemophilia Gene Therapy
Heterogeneous loss of function mutations at F8 and F9 genes causes X-linked recessive bleeding disorders, hemophilia A (HA) and hemophilia B (HB), respectively. HA is clinically indistinguishable from HB and accounts for more than 80% of hemophilia cases; the former affects 1/5000 and the latter 1/25000 male births worldwide. In Iran, it is estimated that around 4300 HA and 900 HB patients are ...
متن کاملTropism-Modification Strategies for Targeted Gene Delivery Using Adenoviral Vectors
Achieving high efficiency, targeted gene delivery with adenoviral vectors is a long-standing goal in the field of clinical gene therapy. To achieve this, platform vectors must combine efficient retargeting strategies with detargeting modifications to ablate native receptor binding (i.e. CAR/integrins/heparan sulfate proteoglycans) and "bridging" interactions. "Bridging" interactions refer to co...
متن کاملGene therapy in Alzheimer’s disease – potential for disease modification
Alzheimer's disease (AD) is the major cause of dementia in the elderly, leading to memory loss and cognitive decline. The mechanism underlying onset of the disease has not been fully elucidated. However, characteristic pathological manifestations include extracellular accumulation and aggregation of the amyloid beta-peptide (Abeta) into plaques and intracellular accumulation and aggregation of ...
متن کاملGenetic modification and genetic determinism
In this article we examine four objections to the genetic modification of human beings: the freedom argument, the giftedness argument, the authenticity argument, and the uniqueness argument. We then demonstrate that each of these arguments against genetic modification assumes a strong version of genetic determinism. Since these strong deterministic assumptions are false, the arguments against g...
متن کاملذخیره در منابع من
با ذخیره ی این منبع در منابع من، دسترسی به آن را برای استفاده های بعدی آسان تر کنید
ژورنال
عنوان ژورنال: Experimental Dermatology
سال: 2021
ISSN: 0906-6705,1600-0625
DOI: 10.1111/exd.14314