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[PERSPECTIVES] Emerging Therapies for Breast Cancer
We describe promising examples of novel therapies that have been discovered within each of these paradigms and suggest how future drug development efforts might benefit from the continued application of these principles. (Source: Cold Spring Harbor perspectives in medicine)
Source: Cold Spring Harbor perspectives in medicine - August 1, 2023 Category: Research Authors: Goel, S., Chandarlapaty, S. Tags: Breast Cancer: From Fundamental Biology to Therapeutic Strategies PERSPECTIVES Source Type: research

[PERSPECTIVES] iPSC-RPE in Retinal Degeneration: Recent Advancements and Future Perspectives
Regenerative medicine is a great hope for patients suffering from diseases for which no effective treatment is available. With the creation of induced pluripotent stem cells (iPSCs) in 2006, research and development has accelerated expeditiously, reaching a practical stage worldwide. The iPSC-regenerative medicine in ophthalmology is one of the pioneers, which has kicked off clinical application ahead of other fields owing to its advantages. The clinical safety issues of iPSC-derived retinal pigment epithelial (iPSC-RPE) transplantation for exudative age-related macular degeneration have been addressed to a certain extent....
Source: Cold Spring Harbor perspectives in medicine - August 1, 2023 Category: Research Authors: Maeda, T., Takahashi, M. Tags: Retinal Disorders: Genetic Approaches to Diagnosis and Treatment PERSPECTIVES Source Type: research

[PERSPECTIVES] Gut Microbiota and the Microvasculature
The gut microbiota is increasingly recognized as an actuating variable shaping vascular development and endothelial cell function in the intestinal mucosa but also affecting the microvasculature of remote organs. In the small intestine, colonization with gut microbiota and subsequent activation of innate immune pathways promotes the development of intricate capillary networks and lacteals, influencing the integrity of the gut–vascular barrier as well as nutrient uptake. Since the liver yields most of its blood supply via the portal circulation, the hepatic microcirculation steadily encounters microbiota-derived patte...
Source: Cold Spring Harbor perspectives in medicine - August 1, 2023 Category: Research Authors: Kiouptsi, K., Pontarollo, G., Reinhardt, C. Tags: Angiogenesis PERSPECTIVES Source Type: research

[PERSPECTIVES] Prevention of Epithelial Ovarian Cancer
Given the challenges with achieving effective and durable treatment for epithelial ovarian cancer, primary prevention is highly desirable. Fortunately, decades of research have provided evidence for several strategies that can be deployed to optimize risk reduction. These include surgery, chemoprevention, and lifestyle factor modifications. These broad categories vary in terms of the magnitude of risk reduction possible, the possible short-term and long-term side effects, the degree of difficulty, and acceptability. Thus, the concept of a risk-based model to personalize preventive interventions is advocated to guide discus...
Source: Cold Spring Harbor perspectives in medicine - August 1, 2023 Category: Research Authors: Sellers, T. A., Peres, L. C., Hathaway, C. A., Tworoger, S. S. Tags: Ovarian Cancer PERSPECTIVES Source Type: research

[PERSPECTIVES] High-Throughput Identification, Modeling, and Analysis of Cancer Driver Genes In Vivo
The vast number of genomic and molecular alterations in cancer pose a substantial challenge to uncovering the mechanisms of tumorigenesis and identifying therapeutic targets. High-throughput functional genomic methods in genetically engineered mouse models allow for rapid and systematic investigation of cancer driver genes. In this review, we discuss the basic concepts and tools for multiplexed investigation of functionally important cancer genes in vivo using autochthonous cancer models. Furthermore, we highlight emerging technical advances in the field, potential opportunities for future investigation, and outline a visi...
Source: Cold Spring Harbor perspectives in medicine - July 5, 2023 Category: Research Authors: Tang, Y. J., Shuldiner, E. G., Karmakar, S., Winslow, M. M. Tags: Modeling Cancer in Mice PERSPECTIVES Source Type: research

[PERSPECTIVES] Adaptive Optics Imaging of Inherited Retinal Disease
The human retina is amenable to direct, noninvasive visualization using a wide array of imaging modalities. In the ~140 years since the publication of the first image of the living human retina, there has been a continued evolution of retinal imaging technology. Advances in image acquisition and processing speed now allow real-time visualization of retinal structure, which has revolutionized the diagnosis and management of eye disease. Enormous advances have come in image resolution, with adaptive optics (AO)-based systems capable of imaging the retina with single-cell resolution. In addition, newer functional imaging tech...
Source: Cold Spring Harbor perspectives in medicine - July 5, 2023 Category: Research Authors: Duncan, J. L., Carroll, J. Tags: Retinal Disorders: Genetic Approaches to Diagnosis and Treatment PERSPECTIVES Source Type: research

[PERSPECTIVES] Overview of Retinal Gene Therapy: Current Status and Future Challenges
The success of the first Food and Drug Administration (FDA)- and European Medicines Agency (EMA)-approved gene therapy for genetic disease, voretigene neparovovec-rzyl, (Luxturna) has helped pave the way for development of retinal gene therapies to target other genetic and acquired forms of blindness. Gene therapy trials are now taking place in multiple continents and numerous countries, they use several different gene transfer reagents ("vectors"), studies have used several different routes of administration, and different strategies are being tested in interventional studies with promising results. The future has never b...
Source: Cold Spring Harbor perspectives in medicine - July 5, 2023 Category: Research Authors: Bennett, J. Tags: Retinal Disorders: Genetic Approaches to Diagnosis and Treatment PERSPECTIVES Source Type: research

[PERSPECTIVES] Crowdfunding and Crowdsourcing of Aging Science
Crowdsourcing is a term that describes a method of distributed effort by which a goal is accomplished by a large group of people, typically initiated by a public call-to-action and is a portmanteau of the words "crowd" and "outsourcing" coined in 2005. Crowdfunding is the practice of funding a project or venture via crowdsourced financial contributions. Crowdsourcing and crowdfunding have been used to advance scientific progress for hundreds of years, with the frequency and scale of their use rapidly increasing during the twenty-first century, thanks to the advent of the internet and the mainstream adoption of related conn...
Source: Cold Spring Harbor perspectives in medicine - July 5, 2023 Category: Research Authors: Comito, K. Tags: Aging PERSPECTIVES Source Type: research

[PERSPECTIVES] Embryological Insights into the Origin of Epithelial Cancers of the Female Reproductive Tract
The embryology of the female reproductive organs is reviewed focusing on aspects relevant to the histogenesis of reproductive neoplasms. The evidence reviewed suggests that (1) there is no embryological link between the ovarian surface epithelium and the coelomic epithelium; (2) the ovarian surface epithelium is not composed of pluripotent cells that readily differentiate into various components of the reproductive tract before or after birth; (3) there is no embryological link between the ovarian surface epithelium and the Müllerian ducts, from which internal female reproductive organs other than the ovaries, incl...
Source: Cold Spring Harbor perspectives in medicine - July 5, 2023 Category: Research Authors: Austria, T., Dubeau, L. Tags: Ovarian Cancer PERSPECTIVES Source Type: research

[PERSPECTIVES] Pathomechanisms of Inherited Retinal Degeneration and Perspectives for Neuroprotection
The precise processes causing photoreceptor cell death in retinal degeneration (RD) are still largely unknown but are likely to follow a variety of degenerative mechanisms. While different genetic insults can trigger distinct molecular pathways, eventually these may converge into a limited number of common cell death mechanisms. These mechanisms often involve deregulation of cyclic guanosine monophosphate (cGMP)-signaling and proteostasis, which both may increase photoreceptor energy expenditure. Comprehensive information on these mechanisms may allow for targeted interventions to delay or prevent photoreceptor loss. Here,...
Source: Cold Spring Harbor perspectives in medicine - June 1, 2023 Category: Research Authors: Tolone, A., Sen, M., Chen, Y., Ueffing, M., Arango-Gonzalez, B., Paquet-Durand, F. Tags: Retinal Disorders: Genetic Approaches to Diagnosis and Treatment PERSPECTIVES Source Type: research

[PERSPECTIVES] A Systematic Review of Optogenetic Vision Restoration: History, Challenges, and New Inventions from Bench to Bedside
Blindness due to rod-cone dystrophies is a significant comorbidity and cause of reduced quality of life worldwide. Optogenetics uses adeno-associated viral (AAV) vectors to bypass lost photoreceptors and transfect remnant cell populations of the degenerated retina aiming to restore vision via the ectopic expression of opsins. The optogenetic targeting of retinal ganglion cells (RGCs) has been remarkably successful and several studies have advanced to clinical trials over the recent years. The inner retina and specifically ON bipolar cells represent even more appealing targets due to their intrinsically coded tasks in paral...
Source: Cold Spring Harbor perspectives in medicine - June 1, 2023 Category: Research Authors: Stefanov, A., Flannery, J. G. Tags: Retinal Disorders: Genetic Approaches to Diagnosis and Treatment PERSPECTIVES Source Type: research

[PERSPECTIVES] Mobility Testing and Other Performance-Based Assessments of Functional Vision in Patients with Inherited Retinal Disease
This article reviews performance-based tools developed to assess functional vision, such as the multi-luminance mobility test (MLMT) or the functional low-vision observer-rated assessment (FLORA), and highlights some of the recent advancements used in clinical development for IRD or ocular interventional therapies. (Source: Cold Spring Harbor perspectives in medicine)
Source: Cold Spring Harbor perspectives in medicine - June 1, 2023 Category: Research Authors: Chung, D., Authie, C., Blouin, L. Tags: Retinal Disorders: Genetic Approaches to Diagnosis and Treatment PERSPECTIVES Source Type: research

[PERSPECTIVES] Influence of Aging Science on Global Wealth Management
Modern longevity transforms long-held assumptions about working, saving, investing, and spending—creating both new opportunities and new challenges for wealth management in the 21st century. To adapt to these shifts, our aging world must evolve its strategies and tools to help people maximize their healthy, productive years, work longer and differently beyond traditional retirement age should they choose to do so, manage caregiving responsibilities, and use a range of tools to plan, save, and invest for longer lives. Employers, policymakers, financial services companies, and individuals can all take steps to enable t...
Source: Cold Spring Harbor perspectives in medicine - June 1, 2023 Category: Research Authors: Hodin, M. Tags: Aging PERSPECTIVES Source Type: research

[PERSPECTIVES] Multidimensional Imaging of Breast Cancer
Breast cancer is a pathological condition characterized by high morphological and molecular heterogeneity. Not only the breast cancer cells, but also their tumor micro-environment consists of a multitude of cell types and states, which continuously evolve throughout progression of the disease. To understand breast cancer evolution within this complex environment, in situ analysis of breast cancer and their co-evolving cells and structures in space and time are essential. In this review, recent technical advances in three-dimensional (3D) and intravital imaging of breast cancer are discussed. Moreover, we highlight the resu...
Source: Cold Spring Harbor perspectives in medicine - May 2, 2023 Category: Research Authors: Rios, A. C., van Rheenen, J., Scheele, C. L. G. J. Tags: Breast Cancer: From Fundamental Biology to Therapeutic Strategies PERSPECTIVES Source Type: research

[PERSPECTIVES] Trial by "Firsts": Clinical Trial Design and Regulatory Considerations in the Development and Approval of the First AAV Gene Therapy Product in the United States
Given the therapeutic potential of supplying a normal copy of a mutant gene to the correct target tissue, gene therapy holds extraordinary promise for the treatment of genetic disease. Like other novel classes of therapeutics however, gene therapies must overcome a range of clinical, regulatory, and manufacturing hurdles to reach regulatory approval. This paper reviews key aspects of clinical trial design, development, and evaluation of a novel primary end point, and regulatory interactions that resulted in the first approval by the U.S. Food and Drug Administration (FDA) of an adeno-associated virus (AAV) gene therapy pro...
Source: Cold Spring Harbor perspectives in medicine - May 2, 2023 Category: Research Authors: Reape, K. Z., High, K. A. Tags: Retinal Disorders: Genetic Approaches to Diagnosis and Treatment PERSPECTIVES Source Type: research