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Yale Journal of Biology and Medicine

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https://www.readbyqxmd.com/read/29259533/%C3%A2-regulatory-considerations-for-gene-therapy-products-in-the-us-eu-and-japan%C3%A2
#1
Celine-Lea Halioua-Haubold, James G Peyer, James A Smith, Zeeshaan Arshad, Matthew Scholz, David A Brindley, Robert E MacLaren
Developers of gene therapy products (GTPs) must adhere to additional regulation beyond that of traditional small-molecule therapeutics, due to the unique mechanism-of-action of GTPs and the subsequent novel risks arisen. We have provided herein a summary of the regulatory structure under which GTPs fall in the United States, the European Union, and Japan, and a comprehensive overview of the regulatory guidance applicable to the developer of GTP. Understanding the regulatory requirements for seeking GTP market approval in these major jurisdictions is crucial for an effective and expedient path to market...
December 2017: Yale Journal of Biology and Medicine
https://www.readbyqxmd.com/read/29259532/gene-editing-in-humans-towards-a-global-and-inclusive-debate-for-responsible-research%C3%A2
#2
Itziar de Lecuona, María Casado, Gemma Marfany, Manuel Lopez Baroni, Mar Escarrabill
In December 2016, the Opinion Group of the Bioethics and Law Observatory (OBD) of the University of Barcelona launched a Declaration on Bioethics and Gene Editing in Humans analyzing the use of genome editing techniques and their social, ethical, and legal implications through a multidisciplinary approach. It focuses on CRISPR/Cas9, a genome modification technique that enables researchers to edit specific sections of the DNA sequence of humans and other living beings. This technique has generated expectations and worries that deserve an interdisciplinary analysis and an informed social debate...
December 2017: Yale Journal of Biology and Medicine
https://www.readbyqxmd.com/read/29259531/crispr-patents-and-the-public-health%C3%A2-%C3%A2
#3
Jacob S Sherkow
Patent issues surrounding CRISPR, the revolutionary genetic editing technology, may have important implications for the public health. Patents maintain high prices for novel therapies, limiting patient access. Relatedly, insurance coverage for expensive therapies is waning. Patents also misallocate research and development resources to profitable disease indications rather than those that necessarily impinge on the public health. And it is unclear how CRISPR therapies will figure into the current regulatory framework for biosimilars...
December 2017: Yale Journal of Biology and Medicine
https://www.readbyqxmd.com/read/29259530/%C3%A2-an-appreciation-of-the-gene-an-intimate-history-by-siddhartha-mukherjee-and-a-call-for-expanded-training-in-the-responsible-conduct-of-research%C3%A2
#4
REVIEW
Evan D Morris
The Gene: An Intimate History by Siddhartha Mukherjee, first published in 2016, is a comprehensive and fascinating recounting of the discovery of the gene and genetics research from wrinkled peas to CRISPR/Cas9 and all the details in between. In Mukherjee's sweeping history, the science is clearly depicted but also tightly integrated into the political movements and world events that it spawned, both hopeful and detestable. Two stories from The Gene are the central focuses of this article. One story is driven by the desire of Eugenicists in early 20th century America to rid the population of defective traits...
December 2017: Yale Journal of Biology and Medicine
https://www.readbyqxmd.com/read/29259529/genome-editing-past-present-and-future%C3%A2
#5
REVIEW
Dana Carroll
The CRISPR-Cas genome editing tools have been adopted rapidly in the research community, and they are quickly finding applications in the commercial sector as well. Lest we lose track of the broader context, this Perspective presents a brief review of the history of the genome editing platforms and considers a few current technological issues. It then takes a very limited view into the future of this technology and highlights some of the societal issues that require examination and discussion.
December 2017: Yale Journal of Biology and Medicine
https://www.readbyqxmd.com/read/29259528/%C3%A2-yeast-still-a-beast-diverse-applications-of-crispr-cas-editing-technology-in-s-cerevisiae%C3%A2
#6
REVIEW
Rachael M Giersch, Gregory C Finnigan
The recent discovery and use of CRISPR/Cas9 gene editing technology has provided new opportunities for scientific research in many fields of study including agriculture, genetic disorders, human disease, biotechnology, and basic biological research. The ability to precisely target DNA sequences and either remove, modify, or replace genetic sequences provides a new level of control in nearly all eukaryotic organisms, including budding yeast. Given the many discoveries made in Saccharomyces cerevisiae over the past decades spanning genetics, cell biology, and biochemistry, as well as the development of new technologies that have allowed high throughput screening, robotic automation, and a platform for synthetic genome engineering, the yeast community has also started to recognize the utility and complementary nature of CRISPR-based methodologies...
December 2017: Yale Journal of Biology and Medicine
https://www.readbyqxmd.com/read/29259527/%C3%A2-potential-of-gene-editing-and-induced-pluripotent-stem-cells-ipscs-in-treatment-of-retinal-diseases%C3%A2
#7
REVIEW
Katherine Chuang, Mark A Fields, Lucian V Del Priore
The advent of gene editing has introduced the ability to make changes to the genome of cells, thus allowing for correction of genetic mutations in patients with monogenic diseases. Retinal diseases are particularly suitable for the application of this new technology because many retinal diseases, such as Stargardt disease, retinitis pigmentosa (RP), and Leber congenital amaurosis (LCA), are monogenic. Moreover, gene delivery techniques such as the use of adeno-associated virus (AAV) vectors have been optimized for intraocular use, and phase III trials are well underway to treat LCA, a severe form of inherited retinal degeneration, with gene therapy...
December 2017: Yale Journal of Biology and Medicine
https://www.readbyqxmd.com/read/29259526/science-and-bioethics-of-crispr-cas9-gene-editing-an-analysis-towards-separating-facts-and-fiction%C3%A2
#8
REVIEW
Adam P Cribbs, Sumeth M W Perera
Since its emergence in 2012, the genome editing technique known as CRISPR-Cas9 and its scientific use have rapidly expanded globally within a very short period of time. The technique consists of using an RNA guide molecule to bind to complementary DNA sequences, which simultaneously recruits the endonuclease Cas9 to introduce double-stranded breaks in the target DNA. The resulting double-stranded break is then repaired, allowing modification or removal of specific DNA bases. The technique has gained momentum in the laboratory because it is cheap, quick, and easy to use...
December 2017: Yale Journal of Biology and Medicine
https://www.readbyqxmd.com/read/29259525/%C3%A2-adeno-associated-virus-aav-dual-vector-strategies-for-gene-therapy-encoding-large-transgenes
#9
REVIEW
Michelle E McClements, Robert E MacLaren
The use of adeno-associated viral (AAV) vectors for gene therapy treatments of inherited disorders has accelerated over the past decade with multiple clinical trials ongoing in varying tissue types and new ones initiating every year. These vectors are exhibiting low-immunogenicity across the clinical trials in addition to showing evidence of efficacy, making it clear they are the current standard vector for any potential gene therapy treatment. However, AAV vectors do have a limitation in their packaging capacity, being capable of holding no more than ~5kb of DNA and in a therapeutic transgene scenario, this length of DNA would need to include genetic control elements in addition to the gene coding sequence (CDS) of interest...
December 2017: Yale Journal of Biology and Medicine
https://www.readbyqxmd.com/read/29259524/%C3%A2-genetic-code-expansion-and-optoproteomics
#10
REVIEW
Yuting Chen, Linjie Lu, Shixin Ye
Nature has invented photoreceptor proteins that are involved in sensing and response to light in living organisms. Genetic code expansion (GCE) technology has provided new tools to transform light insensitive proteins into novel photoreceptor proteins. It is achieved by the site-specific incorporation of unnatural amino acids (Uaas) that carry light sensitive moieties serving as "pigments" that react to light via photo-decaging, cross-linking, or isomerization. Over the last two decades, various proteins including ion channels, GPCRs, transporters, and kinases have been successfully rendered light responsive owing to the functionalities of Uaas...
December 2017: Yale Journal of Biology and Medicine
https://www.readbyqxmd.com/read/29259523/%C3%A2-therapeutic-peptide-nucleic-acids-principles-limitations-and-opportunities%C3%A2
#11
REVIEW
Elias Quijano, Raman Bahal, Adele Ricciardi, W Mark Saltzman, Peter M Glazer
Since their invention in 1991, peptide nucleic acids (PNAs) have been used in a myriad of chemical and biological assays. More recently, peptide nucleic acids have also been demonstrated to hold great potential as therapeutic agents because of their physiological stability, affinity for target nucleic acids, and versatility. While recent modifications in their design have further improved their potency, their preclinical development has reached new heights due to their combination with recent advancements in drug delivery...
December 2017: Yale Journal of Biology and Medicine
https://www.readbyqxmd.com/read/29259522/applications-of-crispr-cas9-in-the-mammalian-central-nervous-system%C3%A2-%C3%A2
#12
REVIEW
Katherine E Savell, Jeremy J Day
Within the central nervous system, gene regulatory mechanisms are crucial regulators of cellular development and function, and dysregulation of these systems is commonly observed in major neuropsychiatric and neurological disorders. However, due to a lack of tools to specifically modulate the genome and epigenome in the central nervous system, many molecular and genetic mechanisms underlying cognitive function and behavior are still unknown. Although genome editing tools have been around for decades, the recent emergence of inexpensive, straightforward, and widely accessible CRISPR/Cas9 systems has led to a revolution in gene editing...
December 2017: Yale Journal of Biology and Medicine
https://www.readbyqxmd.com/read/29259521/nuclease-mediated-gene-therapies-for-inherited-metabolic-diseases-of-the-liver%C3%A2-%C3%A2
#13
REVIEW
Taylor E Bryson, Caitlin M Anglin, P Hudson Bridges, Renee N Cottle
Inherited metabolic diseases (IMDs) of the liver represent a vast and diverse group of rare genetic diseases characterized by the loss or dysfunction of enzymes or proteins essential for metabolic pathways in the liver. Conventional gene therapy involving adeno-associated virus (AAV) serotype 8 vectors provide therapeutically high levels of hepatic transgene expression facilitating the correction of the disease phenotype in pre-clinical studies and are currently being evaluated in clinical trials for multiple IMDs...
December 2017: Yale Journal of Biology and Medicine
https://www.readbyqxmd.com/read/29259520/%C3%A2-gene-therapy-for-color-blindness
#14
REVIEW
Mark M Hassall, Alun R Barnard, Robert E MacLaren
Achromatopsia is a rare congenital cause of vision loss due to isolated cone photoreceptor dysfunction. The most common underlying genetic mutations are autosomal recessive changes in CNGA3, CNGB3, GNAT2, PDE6H, PDE6C, or ATF6. Animal models of Cnga3, Cngb3, and Gnat2 have been rescued using AAV gene therapy; showing partial restoration of cone electrophysiology and integration of this new photopic vision in reflexive and behavioral visual tests. Three gene therapy phase I/II trials are currently being conducted in human patients in the USA, the UK, and Germany...
December 2017: Yale Journal of Biology and Medicine
https://www.readbyqxmd.com/read/29259519/%C3%A2-the-application-of-crispr-cas9-for-the-treatment-of-retinal-diseases%C3%A2
#15
REVIEW
Caroline F Peddle, Robert E MacLaren
The CRISPR/Cas9 system of genome editing has revolutionized molecular biology, offering a simple, and relatively inexpensive method of creating precise DNA edits. It has potential application in gene therapy treatment of retinal diseases providing targeted disruption, alteration, or transcriptional regulation of pathogenic genes. In vivo studies have demonstrated therapeutic benefit for a variety of diseases. Despite this, there are many challenges to clinical use of CRISPR/Cas9, including editing efficiency, off-target effects, and disease heterogeneity...
December 2017: Yale Journal of Biology and Medicine
https://www.readbyqxmd.com/read/29259518/%C3%A2-genome-surgery-and-gene-therapy-in-retinal-disorders
#16
REVIEW
Lawrence Chan, Vinit B Mahajan, Stephen H Tsang
The emergence of genome surgery techniques like the clustered regularly interspaced short palindromic repeats (CRISPR) editing technology has given researchers a powerful tool for precisely introducing targeted changes within the genome. New modifications to the CRISPR-Cas system have been made since its recent discovery, such as high-fidelity Cas9 variants to reduce off-target effects and transcriptional activation/silencing with CRISPRa/CRISPRi. The applications of CRISPR-Cas and gene therapy in ophthalmic diseases have been necessary and fruitful, especially given the impact of blinding diseases on society and the large number of monogenic disorders of the eye...
December 2017: Yale Journal of Biology and Medicine
https://www.readbyqxmd.com/read/28955189/the-diabetic-dog-as-a-translational-model-for-human-islet-transplantation%C3%A2
#17
REVIEW
Christopher A Adin, Chen Gilor
The dog model has served as the primary method for early development of many diabetes therapies, including pancreatic islet transplantation techniques and immunosuppressive protocols. Recent trends towards the use of monoclonal antibody therapies for immunosuppression in human islet transplantation have led to the increasing use of primate models with induced diabetes. In addition to induced-disease models in large animals, scientists in many fields are considering the use of naturally-occurring disease models in client-owned pets...
September 2017: Yale Journal of Biology and Medicine
https://www.readbyqxmd.com/read/28955188/premenopausal-reproductive-health-modulates-future-cardiovascular-risk-comparative-evidence-from-monkeys-and-women%C3%A2-%C3%A2-%C3%A2
#18
REVIEW
Jay R Kaplan, Stephen B Manuck
Coronary heart disease (CHD) remains the major cause of mortality among postmenopausal women living in industrialized countries. Several lines of evidence suggest that ovarian hormones (especially estrogen) protect the coronary arteries of premenopausal women. However, it is also known that women commonly experience disruptions in cyclic hormonal function during their reproductive years. In this perspective, we hypothesize that if regular, cyclic ovarian function affords protection against CHD, ovulatory abnormalities in young women may conversely promote the development of atherosclerosis (the pathobiological process underlying CHD) in the years prior to menopause and thus substantially increase the risk of subsequent heart disease...
September 2017: Yale Journal of Biology and Medicine
https://www.readbyqxmd.com/read/28955187/comparative-medicine-an-inclusive-crossover-discipline%C3%A2-%C3%A2-%C3%A2
#19
REVIEW
James Macy, Tamas L Horvath
Comparative Medicine is typically defined as a discipline which relates and leverages the biological similarities and differences among animal species to better understand the mechanism of human and animal disease. It has also been defined as a field of study concentrating on similarities and differences between human and veterinary medicine and is increasingly associated with animal models of human disease, including the critical role veterinarians, animal resource centers, and Institutional Animal Care and Use Committees play in facilitating and ensuring humane and reproducible laboratory animal care and use...
September 2017: Yale Journal of Biology and Medicine
https://www.readbyqxmd.com/read/28955186/extracellular-vesicles-evolutionarily-conserved-mediators-of-intercellular-communication
#20
REVIEW
Charlotte Lawson, Dory Kovacs, Elizabeth Finding, Emily Ulfelder, Virginia Luis-Fuentes
Extracellular vesicles (EV) are sub-micron circulating vesicles found in all bodily fluids and in all species so far tested. They have also recently been identified in seawater and it has further been shown that they are released from microorganisms and may participate in interspecies communication in the gut. EV are typically composed of a lipid bilayer formed from the plasma membrane and which encases a cargo that can include genetic material, proteins, and lipids. At least two different processes of formation and release have been described in mammalian cells...
September 2017: Yale Journal of Biology and Medicine
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