Abstract:
:Inherited epidermolysis bullosa (EB) is a family of rare genetic skin disorders characterized by structural and mechanical fragility of skin and mucosal membranes. The main feature of EB is the presence of recurrent skin blistering or erosions, which have a profound impact in the quality of life of EB patients and, in the most severe forms, cause early lethality. During the past two decades, it became possible to identify mutations in genes responsible for different types of EB and characterize the abnormalities of the related proteins. Nowadays, there is no cure for EB; all the treatments are palliative and focused on the relief of the devastating EB clinical picture. Recent advancements in molecular biology, stem cell biology and regenerative medicine have fostered new therapeutic approaches for EB. This review is focused on recent developments in gene therapy, protein replacement and cell-based therapy for EB, all aimed at finding a cure for this devastating disease.
journal_name
Regen Medjournal_title
Regenerative medicineauthors
Carulli S,Contin R,De Rosa L,Pellegrini G,De Luca Mdoi
10.2217/rme.13.33subject
Has Abstractpub_date
2013-07-01 00:00:00pages
467-81issue
4eissn
1746-0751issn
1746-076Xjournal_volume
8pub_type
杂志文章,评审abstract::Genetics Policy Institute (GPI) is a nonprofit, public interest organization recognized as a leading promoter and defender of stem cell research and other cutting-edge medical research targeting cures. Since GPI's inception in 2003, its mission has focused on establishing a positive policy, regulatory and societal fra...
journal_title:Regenerative medicine
pub_type: 新闻
doi:10.2217/rme.09.6
更新日期:2009-05-01 00:00:00
abstract::Many of the current drug therapies for androgenetic alopecia were discovered serendipitously, with hair growth observed as an off-target effect when drugs were used to treat a different disorder. Subsequently, several studies using cultured cells have enabled identification of hair growth modulators with similar prope...
journal_title:Regenerative medicine
pub_type: 杂志文章,评审
doi:10.2217/rme.13.87
更新日期:2014-01-01 00:00:00
abstract::Background: To evaluate whether subtenon injection of platelet-rich plasma (PRP) with retinal electromagnetic stimulation (rEMS) is effective in therapy-resistant chronic central serous chorioretinopathy (CSCR). Design: Prospective, sequential. Materials & methods: The study included 22 eyes with resistant chronic CSC...
journal_title:Regenerative medicine
pub_type: 杂志文章
doi:10.2217/rme-2020-0056
更新日期:2020-08-01 00:00:00
abstract::Tendons and ligaments are related connective tissues that join muscle to bone and bone to bone, respectively. Tendon and ligament injuries are widely distributed clinical problems in society and while healing of such disorders can occur, the original biological properties of the tissue do not return to normal. In this...
journal_title:Regenerative medicine
pub_type: 杂志文章,评审
doi:10.2217/17460751.1.4.563
更新日期:2006-07-01 00:00:00
abstract:AIM:To perform an early cost-effectiveness analysis of in vitro expanded myoblasts (IVM) and minced myofibers versus midurethral slings (MUS) for surgical treatment of female stress urinary incontinence. METHODS:Cost-effectiveness and sensitivity analyses were performed using a decision tree comprising previously publ...
journal_title:Regenerative medicine
pub_type: 杂志文章
doi:10.2217/rme-2017-0124
更新日期:2018-04-01 00:00:00
abstract::Aim: Cell repopulation of tissue-engineered vascular grafts (TEVGs) from decellularized arterial scaffolds is limited by dense concentric tunica media layers which impede cells migrating radially between the layers. We aimed to develop and validate a new microneedle device to modify decellularized carotid arteries wit...
journal_title:Regenerative medicine
pub_type: 杂志文章
doi:10.2217/rme-2019-0011
更新日期:2019-11-01 00:00:00
abstract::Promising new medical technologies and approaches to treatment offer the potential to substantially improve the lives of patients who need help, and in some cases transform the way medicine is practiced. In recent years, the biotechnology industry has generated many examples of new approaches that are having a profoun...
journal_title:Regenerative medicine
pub_type: 杂志文章
doi:10.2217/rme.12.90
更新日期:2012-11-01 00:00:00
abstract::Aim: The present study evaluated the effects of polydeoxyribonucleotide (PDRN) on tissue regeneration, paying special attention to the molecular mechanisms that underlie its tissue remodeling actions to better identify its effective therapeutic potential in wound healing. Materials & methods: Strategic searches were c...
journal_title:Regenerative medicine
pub_type: 杂志文章
doi:10.2217/rme-2019-0118
更新日期:2020-08-06 00:00:00
abstract:AIM:It is unknown if the beneficial effects of mesenchymal stromal cells (MSC) transplantation into the liver are dependent on their anchorage and differentiation into hepatocytes or rather the result of the release of stem cell intracellular content with hepatoprotector properties. MATERIALS & METHODS:The effects of ...
journal_title:Regenerative medicine
pub_type: 杂志文章
doi:10.2217/rme.14.59
更新日期:2015-01-01 00:00:00
abstract:AIM:Dendritic cell (DC)-based vaccines have a potential utility for use in the treatment of malignancy. Human embryonic stem cells (hESCs) may provide a more cost-effective and reliable source of DCs for immunotherapy purposes, providing on-demand access for patients. METHOD:We developed a protocol to generate DCs fro...
journal_title:Regenerative medicine
pub_type: 杂志文章
doi:10.2217/rme.09.25
更新日期:2009-07-01 00:00:00
abstract::Hematopoietic stem cells (HSCs) have been shown recently to hold much promise in curing autoimmune diseases. Newly diagnosed Type 1 diabetes individuals have been successfully reverted to normoglycemia by administration of autologous HSCs in association with a nonmyeloablative regimen (antithymocyte globulin + cycloph...
journal_title:Regenerative medicine
pub_type: 杂志文章,评审
doi:10.2217/rme-2015-0057
更新日期:2016-06-01 00:00:00
abstract::Owing to the complicated and time-consuming regenerative process, the repair of injured peripheral nerves depends largely on ongoing stem-cell therapy. Decades ago, researchers successfully isolated and identified muscle-derived stem cells (MDSCs) and discovered their potential for multidifferentiation. MDSCs play an ...
journal_title:Regenerative medicine
pub_type: 杂志文章,评审
doi:10.2217/rme-2016-0165
更新日期:2017-04-01 00:00:00
abstract:AIM:To evaluate the cost-effectiveness of autologous cell therapy manufacturing in xeno-free conditions. MATERIALS & METHODS:Published data on the isolation and expansion of mesenchymal stem/stromal cells introduced donor, multipassage and culture media variability on cell yields and process times on adherent culture ...
journal_title:Regenerative medicine
pub_type: 杂志文章
doi:10.2217/rme-2018-0034
更新日期:2018-12-01 00:00:00
abstract::Worldwide, tissue engineering and cellular replacement therapies are at the forefront of the regenerative medicine agenda, and researchers are addressing key diseases, including diabetes, stroke and neurological disorders. It is becoming evident that neurological cell therapy is a necessarily complex endeavor. The bra...
journal_title:Regenerative medicine
pub_type: 杂志文章,评审
doi:10.2217/rme.12.2
更新日期:2012-05-01 00:00:00
abstract::Aim: We aimed to evaluate the capacity of the bilayer polylactic-co-glycolic acid (PLGA)/TGF-β3/adipose-derived mesenchymal stem cell (ADSC) construct used to repair cartilage defects and the role of ADSCs in the repair process in vivo. Materials & methods: Defects were created surgically on the femoropatellar groove ...
journal_title:Regenerative medicine
pub_type: 杂志文章
doi:10.2217/rme-2019-0151
更新日期:2020-06-01 00:00:00
abstract::Mesenchymal stem cells (MSCs) have tremendous potential for regenerative medicine, and have been researched for the treatment of cardiovascular diseases. MSCs are a promising cell type because of their ease of isolation and expansion, their multipotency and their low immunogenicity. However, in order to fully utilize ...
journal_title:Regenerative medicine
pub_type: 杂志文章,评审
doi:10.2217/17460751.3.6.877
更新日期:2008-11-01 00:00:00
abstract::Biocompatibility is key to the performance of any material in a biological environment. This review outlines current opinion on the factors that lead to biocompatibility and focuses on the interactions that occur at the interface between material and environment. The sequence of events, from protein adsorption, cell a...
journal_title:Regenerative medicine
pub_type: 杂志文章,评审
doi:10.2217/17460751.1.6.789
更新日期:2006-11-01 00:00:00
abstract:AIM:We hypothesized that delivery of mesenchymal stem cells (MSCs) in a biomimetic collagen scaffold improves wound healing in a diabetic mouse model. MATERIALS & METHODS:Rolled collagen scaffolds containing MSCs were implanted or applied topically to diabetic C57BL/6 mice with excisional wounds. RESULTS:Rolled scaff...
journal_title:Regenerative medicine
pub_type: 杂志文章
doi:10.2217/rme-2015-0045
更新日期:2016-04-01 00:00:00
abstract:AIMS:To evaluate the safety and efficacy of cell therapies administered acutely/sub-acutely after stroke. METHODS:Five databases were searched for studies examining the safety/efficacy of cell therapies administered ≤90 days post-stroke. Reporting quality and adherence to research guidelines were evaluated. Safety and...
journal_title:Regenerative medicine
pub_type: 杂志文章,meta分析,评审
doi:10.2217/rme-2016-0063
更新日期:2016-10-01 00:00:00
abstract::Finding solutions for patients with unmet needs is at the heart of Genzyme's innovative medical research. While small molecules and enzyme-replacement therapies have made significant strides in improving patients' quality of life and expectancy, the solutions to many conditions, such as heart disease or CNS diseases, ...
journal_title:Regenerative medicine
pub_type: 杂志文章
doi:10.2217/17460751.2.1.95
更新日期:2007-01-01 00:00:00
abstract::Many features of adipose stem/progenitor cells, including their physiological functions and localization, have been clarified in the past decade. Adipose tissue turns over very slowly, with perivascular progenitor cells differentiating into new adipocytes to replace dead adipocytes. A number of clinical trials using f...
journal_title:Regenerative medicine
pub_type: 杂志文章,评审
doi:10.2217/rme.11.62
更新日期:2011-11-01 00:00:00
abstract:AIM:Glucotoxicity obstructs pancreatic differentiation from adult stem cells. The aim was to develop a novel protocol for differentiation of dental pulp stem cells (DPSCs) into pancreatic β cells and determine the effect of H2S on glucotoxicity. MATERIALS & METHODS:DPSCs were differentiated with media containing 5.5 o...
journal_title:Regenerative medicine
pub_type: 杂志文章
doi:10.2217/rme-2016-0142
更新日期:2017-03-01 00:00:00
abstract::Tissue engineering is a field of study unto itself, but in reality, it is a fusion of medicine, pharmacology, chemistry, cell biology, molecular biology and engineering. The field has been developing at an ever-increasing pace and already provides benefits to regenerative medicine in areas such as the skin and cornea....
journal_title:Regenerative medicine
pub_type: 杂志文章,评审
doi:10.2217/rme.13.16
更新日期:2013-05-01 00:00:00
abstract::Regenerative medicine is an evolving, cross-disciplinary, international field that, as a result, uses terms that are either not widely understood, or may have a number of different meanings. Many stakeholders have identified this lack of clarity as a potential barrier to effective communication within the field. To ad...
journal_title:Regenerative medicine
pub_type: 杂志文章
doi:10.2217/rme.09.34
更新日期:2009-07-01 00:00:00
abstract::Regenerative engineering has been defined as the convergence of Advanced Materials Sciences, Stem Cell Sciences, Physics, Developmental Biology and Clinical Translation for the regeneration of complex tissues and organ systems. Anterior cruciate ligament (ACL) reconstruction necessitates the regeneration of bone, liga...
journal_title:Regenerative medicine
pub_type: 杂志文章,评审
doi:10.2217/rme-2016-0125
更新日期:2016-12-01 00:00:00
abstract::Founded in 2009 and headquartered in Glasgow, Scotland, UK, Sistemic Ltd has developed from a thought in the minds of four scientists into a company working globally to play its part in delivering the exciting opportunities for improvements in human health presented by cell therapies and regenerative medicine products...
journal_title:Regenerative medicine
pub_type: 杂志文章
doi:10.2217/rme.13.58
更新日期:2013-09-01 00:00:00
abstract::The concept of stem cell therapy has engaged the attention of the public and scientists alike. Intensive research effort is focused upon understanding the biology and therapeutic potential of embryonic and adult stem cells, with the eventual goal of treating such pathologies as Parkinson's disease, diabetes, neurologi...
journal_title:Regenerative medicine
pub_type: 杂志文章
doi:10.2217/17460751.1.5.715
更新日期:2006-09-01 00:00:00
abstract::Dimethyl sulfoxide (DMSO) is the cryoprotectant of choice for most animal cell systems since the early history of cryopreservation. It has been used for decades in many thousands of cell transplants. These treatments would not have taken place without suitable sources of DMSO that enabled stable and safe storage of bo...
journal_title:Regenerative medicine
pub_type: 杂志文章
doi:10.2217/rme-2019-0145
更新日期:2020-03-01 00:00:00
abstract::Aim: To determine the potential of mesenchymal stem cells (MSC) for corneal epithelial regeneration in vitro. Materials & methods: Bone marrow MSC (BM-MSC) and adipose tissue MSC were analyzed for corneal epithelial and mesenchymal markers, using limbal stem cells and corneal cells as controls. MSC with better potenti...
journal_title:Regenerative medicine
pub_type: 杂志文章
doi:10.2217/rme-2019-0067
更新日期:2020-03-01 00:00:00
abstract:OBJECTIVES:Mechanisms underpinning Gram-negative bacterial vaginosis-induced birth anomalies are obscure. Ethical issues limit such studies on peri-implantation-stage human embryos. Here we have used embryoid bodies (EBs) as an in vitro model to examine the effect of Gram-negative bacterial endotoxins/lipopolysaccharid...
journal_title:Regenerative medicine
pub_type: 杂志文章
doi:10.2217/17460751.3.1.23
更新日期:2008-01-01 00:00:00