From Bench to Bedside: FDA Approves First CRISPR-based Gene Therapy

Written by: Madison Fitzgerald

Edited by: Ryan Schildcrout, Jennifer Baker, Christina Del Greco, Ari Hoffman, and Emma Milligan

Illustrated by: Zoey Yeoh

We inherit a lot of things from our parents–an old jean jacket, family recipes, or even a penchant for dessert. On the cellular level, we inherit a set of genes from each of our parents that determine traits like eye color and blood type. In some unfortunate cases, people can inherit genes that cause disease. Most treatments that are currently available for genetic diseases help manage symptoms but are not curative because the disease-causing gene remains broken.

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La Genética y la Ilusión de Precisión

Autor: Christa Ventresca

Editores: Christina Del Greco, Andres Rivera Ruiz, Kate Giffin, and Jennifer Baker

Ilustración: Saaj Chattopadhyay

Traducción: Llilian Arzola Martínez y Rocío Cisneros 

Esta es la primera parte de una serie conformada por tres blogs que exploran el impacto de los análisis genéticos en la identidad personal. ¡Pronto publicarémos la segunda y tercera parte!

Si tienes curiosidad por descubrir la información que se oculta dentro de tu ADN, hoy en día disponemos de la tecnología necesaria para explorar nuestros genes. Puede ser que desees verificar el historial familiar de tus ancestros, o tal vez te preocupe la presencia de enfermedades genéticas en tu familia. Para obtener más información al respecto, puedes enviar una muestra de saliva a la empresa popular de análisis genéticos “23andMe”. Los resultados que recibirás incluirán una serie de estadísticas y números, todos ellos fundamentados en tu ADN. Sin embargo, ¿cómo interpretarás estos resultados? Y, ¿cuánta confianza tendrás en que todos los datos sean precisos?

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Decoding Herculaneum Scrolls with Artificial Intelligence

Written by: Julia Donovan

Edited by: Madison Fitzgerald, Kapil Shrawankar, Nick Janne, Ryan Schildcrout

When Mount Vesuvius erupted, it simultaneously destroyed the entire civilization of Pompeii whilst preserving the city under volcanic ash. Among the items excavated in 1752 was a collection of 1,800 scrolls from the nearby city of Herculaneum. The Herculaneum Scrolls are the only known large-scale library in classic antiquity. Given the small number of classical works that have survived beyond the period–Sophocles wrote 120 plays but only 7 remain–there is hope that these scrolls contain unknown works. Some researchers argue that only the best works from antiquity had a chance at survival, meaning the 7 plays of Sophocles that exist were his most popular ones. Evidence for this theory includes the fact that the Iliad was the most copied poem during antiquity, with many private manuscripts of the poem surviving to this day. Other experts argue that the survival of classical works is purely due to chance, supported by the fact that the poems by Catullus survive in only one manuscript. Similarly, the works of Sappho, which were highly regarded in her own times, exist only in fragments. Decoding the contents of these surviving scrolls could extensively add to the body of classical works. However, previous attempts to open the scrolls have led to their destruction. Due to the volcanizing of the scrolls, the brittleness of the papyrus causes breakage and the ink often fades when exposed to air. As a result, approximately 1,000 of the Herculaneum Scrolls remain intact. Researchers began to wonder if there was a way to see inside the scrolls without opening them, and if artificial intelligence (AI) could then help decode what was written. In 2023, AI aided in the discovery of the first word from a Herculaneum scroll. The path to this monumental step built on the AI research of many different labs.    

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关于“意识”的神经科学研究:历史回溯

作者 (Author): 瑞秋·沃尔伯格 (Rachel Wahlberg)

英文编辑 (English Editors): 奥利维亚·皮弗·阿尔(Olivia Pifer Alge), 奥斯汀·香农(Austin Shannon), 安德烈斯·里维拉·鲁伊斯 (Andrés Rivera Ruiz), 詹妮弗·贝克(Jennifer Baker )

插图 (Illustrator): 哈娜·帕斯·哈布曼 (Hana Paz Harbman)

中文翻译 (Chinese Translator): 李易为 (Yiwei Li)

中文编辑 (Chinese Editor): 杨知颖 (Zhiying Yang)

注:本博文是我们有关“意识”的神经科学研究介绍的第一篇。请继续期待我们的第二篇和第三篇!

如果你我在路上相遇,而我问你是否有意识,你会如何回答?我猜你会说:“嗯,有吧?”——如果我们是同一类人,那你可能会像我一样在回答后紧张地笑笑,思考自己方才到底是进行了一段什么样的对话。

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Dr. Leemor Joshua-Tor: Mad about U: Regulating Let7 Pre-miRNA

Live Blogger: Rachael Baliira
Editors: Madison Fitzgerald and Ryan Schildcrout

This piece was written live during the 8th annual RNA Symposium, “Unmasking the Power of RNA: From Structure to Medicine” hosted by the University of Michigan’s Center for RNA Biomedicine. Follow MiSciWriters’ coverage of this event on Twitter with the hashtag #umichrna.

When you look back at your baby photo, first grade school picture with missing teeth, or even your prom photo, and compare them to your latest selfie, have you stopped to marvel at how you’ve developed into the person you are now? If you dive into the mechanisms that ensure normal development, cell differentiation and fate, you would be amazed at all that has to go right to make you ‘you.’ Your body achieves this through gene silencing, which is a negative feedback mechanism that regulates gene expression to define cell fate and timely gene expression. Micro-RNAs (miRNA) are the small agents that carry out gene silencing. They suppress unwanted expression of specific genes by binding to their mature transcripts, known as messenger RNA (mRNA), so that the cell only expresses proteins that are needed at the time. When this happens, the marked mRNA are destroyed instead of being translated into a protein. Thus, miRNA-dependent silencing of gene expression is essential for normal development and cell differentiation states. Over the years, scientists studying miRNA have uncovered much about miRNA-dependent gene silencing activity but there is still much to understand about how miRNAs come to be. Luckily, miRNA synthesis and regulation is a matter of great importance to the Joshua-Tor lab. 

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Dr. Brenton R. Graveley: A Comprehensive Binding and Functional Map of Human RNA-Binding Proteins

Live Blogger: Madison Fitzgerald
Editors: Lirong Shi and Ryan Schildcrout

This piece was written live during the 8th annual RNA Symposium, “Unmasking the Power of RNA: From Structure to Medicine” hosted by the University of Michigan’s Center for RNA Biomedicine. Follow MiSciWriters’ coverage of this event on Twitter with the hashtag #umichrna.

For millennia, humans have been collecting and compiling information. Literally – the first encyclopedia was published in the 1st Century by Pliny the Elder, a Roman statesman. Our funny-named friend compiled 37 chapters worth of information on topics such as astronomy, botany, geology, pharmacology, zoology, and human physiology. In the modern era, scientists look to other encyclopedic sources to find information. We go to PubMed to read journal articles, to GenBank or BV-BRC to view sequenced genomes, and to Kegg Pathway to browse metabolic pathways found in our favorite species. We use these databases containing information from disparate sources to inform our research and facilitate scientific discovery. University of Connecticut Professor Dr. Brenton R. Graveley and his team take a different approach. Rather than compiling data from experiments performed using a variety of materials, methods, and data analysis pipelines, this consortium is generating data with standardized protocols. The end goal? A comprehensive encyclopedia of RNA elements (ENCORE). In his talk at the Center for RNA Biomedicine’s 2024 RNA Symposium, Dr. Graveley presented the progress towards this goal. But first, what are functional RNA elements? 

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Dr. Peter Todd: Short Tandem Repeats in Neuronal Function and Neurological Disease

Live Blogger: Ryan Schildcrout
Editor: Madison Fitzgerald

This piece was written live during the 8th annual RNA Symposium, “Unmasking the Power of RNA: From Structure to Medicine” hosted by the University of Michigan’s Center for RNA Biomedicine. Follow MiSciWriters’ coverage of this event on Twitter with the hashtag #umichrna.

When we think of “DNA,” we normally think about genes that encode proteins. However, the vast majority of the human genome is thought to be “non-coding,” in that the DNA does not encode proteins. Non-coding DNA has been long thought of as biologically inert, but in the last few decades, scientists have started exploring its purpose. Since then, it has been recognized to be a key element in regulating gene expression. Within those non-coding regions exist millions of Short Tandem Repeats, or  microsatellites, which are repetitive DNA sequences of up to 6 base pairs. The number of repeats within a microsatellite can vary drastically across the genome. We know very little about the function of these microsatellites because next-generation sequencing (NGS) technologies are unable to sequence highly repetitive regions of DNA. Some of these repeats have been linked to neurological disorders such as Autism, ALS, and dementia, thus highlighting the need to study the mechanisms by which they cause disease. Dr. Peter Todd from the University of Michigan has recently uncovered some of these mechanisms in context with neurological diseases. 

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Dr. Victoria D’Souza: Structure-based redefinition of the HIV-1 reverse transcription initiation

Live Blogger: Brenna Saladin
Editors: Varsha Shankar and Ryan Schildcrout

This piece was written live during the 8th annual RNA Symposium, “Unmasking the Power of RNA: From Structure to Medicine” hosted by the University of Michigan’s Center for RNA Biomedicine. Follow MiSciWriters’ coverage of this event on Twitter with the hashtag #umichrna.

Viruses are a routine occurrence in everyday life. The common cold, the flu, and most recently the COVID-19 pandemic has kept viruses at the forefront of the public mind. One of the key aspects of the viral life cycle and infectious mechanism is the fact that they rely on the host in order to replicate and spread infection. Viruses do not contain their own machinery for replication, but rather they hijack host cell machinery to do their bidding. Our speaker today studies one of the beginning steps of this process: reverse transcription initiation. This is the process by which the retroviruses, such as human immunodeficiency virus (HIV), create the DNA template from viral RNA that encodes the necessary components for infection and proliferation in host cells. The D’ Souza lab studies these processes so that we have a better understanding of the mechanisms of viral infection, and therefore the potential to create drugs to combat them.

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Dr. Drew Weissman: Nucleoside Modified mRNA-LNP Therapeutics

Live Blogger: Matthew Blacksmith
Editors: Sadie Schaus and Ryan Schildcrout

This piece was written live during the 8th annual RNA Symposium, “Unmasking the Power of RNA: From Structure to Medicine” hosted by the University of Michigan’s Center for RNA Biomedicine. Follow MiSciWriters’ coverage of this event on Twitter with the hashtag #umichrna.

What do measles, the flu, and most recently, COVID-19 have in common? Each of these viruses has vaccines which have been developed to prevent infection, reduce the severity of symptoms, and promote faster recovery. So what are vaccines and how do they work? The answer varies from vaccine to vaccine. Historically, vaccines have been created by weakening or outright killing viruses before injecting them into patients to expose them to viral proteins. Once injected, the body attacks the virus, priming the immune system for a later date where you’re exposed to the virus at full strength. However, the process of creating a vaccine from a weakened virus can be long and difficult. Fortunately, scientists have been hard at work trying to create vaccines which are as effective as the current vaccine standard, but can be produced more quickly in response to global needs.

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La jornada agotadora de 60 horas: Desafíos y Oportunidades al Trabajar con el Láser de Electrones Libres de Rayos X 

Escrito por: Eilidh McClain
Editado por: Olivia Pifer Alge, Mena Davidson, Kristen Loesel, y Jennifer Baker
Ilustrado por: Jacquelyn Roberts
Traducido por: Juan Blume La Torre y Rocío Cisneros 

Inicio del experimento. Turno uno. 3 horas transcurridas.

¡Cucú! “La fase de escaneo ha concluido”.

Desde la sala de control en las instalaciones del Láser de Electrones Libres de Rayos X Europeo (XFEL) en Hamburgo, Alemania, pudimos escuchar el anuncio de que el escaneo ya había terminado. La primera vez que escuché el anuncio, no lo esperaba; es un sonido tan sutil que fácilmente podría pasar desapercibido e insignificante, lo cual contrasta notablemente con la importancia del laboratorio científico. Aunque inicialmente inesperado, este sonido se volvió un ritual familiar de celebración, marcando cada triunfo en nuestra serie de experimentos.

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