#He Jiankui

[wisedreamlandlove]

Umstrittener Genforscher schlägt neues Experiment zur Vorbeugung der Alzheimer-Krankheit vor

Der chinesische Wissenschaftler, der wegen der Veränderung der Gene von Babys weltweit in die Kritik geraten ist, möchte mit einer ähnlichen Technik der Alzheimer-Krankheit vorbeugen.

He Jiankui, der 2018 die Welt mit der Ankündigung schockierte, er habe die Gene von Embryonen verändert, um sie resistent gegen HIV zu machen, schlägt nun vor zu testen, ob eine bestimmte genetische Mutation Schutz gegen die häufigste Ursache von Demenz bietet. Bei der Studie würden keine menschlichen Embryonen eingepflanzt, um eine Schwangerschaft herbeizuführen - was bedeutet, dass keine Babys geboren werden - und sie wird zunächst an Mäusen erprobt, wie er Ende letzter Woche auf seinem Twitter-Account mitteilte.

Es ist unklar, wie das Experiment ablaufen würde, da behördliche und ethische Genehmigungen erforderlich sind und er ein lebenslanges Verbot für die Arbeit in der Reproduktionstechnologie verbüßt. Er sagte, dass die genetische Mutation, die er in die Embryonen einführen will, die Bildung einer Plaque im Gehirn, die das Kennzeichen von Alzheimer ist, verringern würde.

Nach seiner Entlassung aus dem Gefängnis im Jahr 2022, als er eine dreijährige Haftstrafe für sein früheres Experiment abgesessen hatte, tauchte er wieder in China auf, um ein neues Labor zu gründen. Diese Arbeit - die im Geheimen durchgeführt und erst nach der Geburt von Zwillingsmädchen enthüllt wurde - wurde als unverantwortlicher Einsatz einer Technologie angeprangert, deren langfristige Auswirkungen kaum bekannt sind.

Obwohl sich die Gentechnologie bei genetischen Störungen und seltenen Krankheiten als nützlich erwiesen hat, unterliegt sie strengen Vorschriften. Die meisten Länder, darunter auch China, verbieten die Veränderung von Genen in menschlichen Embryonen, da die Veränderungen an künftige Generationen weitergegeben werden könnten, was möglicherweise unbeabsichtigte Folgen hätte.

Über das Leben der Kinder, die 2018 als Ergebnis von He's Experiment geboren wurden, ist wenig bekannt. Nach der Kontroverse kündigte China an, klinische Versuche mit Genmanipulation und anderen experimentellen biowissenschaftlichen Technologien strenger zu kontrollieren.

Die frühere Arbeit von He Jiankui stützte sich auf die CRISPR-Genbearbeitung, obwohl spätere Studien gezeigt haben, dass die Technologie auch das Risiko birgt, genetisches Material zu beschädigen und schwerwiegende Nebenwirkungen in den Zellen menschlicher Embryonen zu verursachen. In seinem jüngsten Vorschlag wies er auf die Risiken von CRISPR hin und erklärte, dass sein neuer Ansatz das Base Editing verwenden wird, das seiner Meinung nach sicherer ist.

Hunderte von Milliarden Dollar haben die Arzneimittelhersteller weltweit für die Behandlung von Alzheimer ausgegeben, aber es gibt derzeit kein Heilmittel für diese Krankheit.

[watercress-words]

THE MUTANT PROJECT-a book review

I review Dr. Eben Kirksey's book The Mutant Project-inside the global race to genetically modify humans

THE MUTANT PROJECT Inside the global race to genetically modify humans By Eben Kirksey If this were the title of a fiction book, you might expect the main characters to be brilliant but misguided scientists, funded by biotech start-ups who see potential for massive profits by developing methods to manipulate human DNA. The applications of such technology for the treatment of genetic diseases,…

[aorish]

scientist Jiankui He stunned—and dismayed—the genetic community when he announced he had already used CRISPR, which many believe still hasn’t been proven either safe or effective in human patients, to permanently alter the genomes of twin girls to be immune to HIV infection. He’s experiment was criticized because he edited the twins’ cells when they were embryos, therefore ensuring that every one of their cells is now changed, including their reproductive ones, which means their edited genomes can be passed on to their children, despite the fact that experts can’t be sure what the long term effects of such lasting modifications might be.

maybe the e/acc people have the right of it actually

[aurpiment]

hey i just saw your rb of the jiankui he tweet and he didn’t actually manage to make the twins immune to hiv - the mutation is a 32bp deletion and you have to have it in both copies of the gene for it to confer immunity (but there’s evidence that it’s not 100%) and his results were a 15bp deletion in one copy for one of the twins and a 4bp deletion in both copies for the other, so not the mutation that confers HIV immunity. it’s unclear what effect these mutations would have, if any. just wanted to clarify bc i’m a biologist haha

Aw man

[she-is-ovarit]

Remember that one time when a Chinese scientist went rogue and effectively edited the genes of three female embryos (two of them twins, now age 4) using CRISPR technology?

[yourmoonmomma]

if anyone has found a copy of the newest documentary on He Jiankui, pls send it to me

[superurmet1]

El creador de los primeros humanos modificados genéticamente lanza un desafío

[biotech-news-feed]

#BioTech #science

[monterplant]

The Download: the future of CRISPR babies, and investing in climate tech

This is today’s edition of The Download, our weekday newsletter that provides a daily dose of what’s going on in the world of technology. Controversial CRISPR scientist promises “no more gene-edited babies” until society comes around He Jiankui, the Chinese biophysicist whose controversial 2018 experiment led to the birth of three gene-edited children, says he’s returned to… Continue reading The…

[tritonmarketresearchamey]

Latest Advances in Gene and Cell Therapies Transform Healthcare

Gene and cell therapies represent a ground-breaking advancement in medical science, offering potential cures for a variety of previously untreatable diseases. These therapies are revolutionizing how we provide targeted healthcare by modifying genetic material or using cells to restore or alter biological functions. Early interventions in congenital disorders can significantly reduce long-term health complications, offering a healthier start to life for newborns. Thus, the potential of gene and cell therapies to transform medical treatments is immense, especially in the field of natal and prenatal care.

A notable example of gene therapy involved the birth of the first babies with edited genes. In 2018, Dr. Jiankui announced the birth of twin girls whose genes were edited using CRISPR technology. He edited and deactivated a gene known as CCR5 with the goal of conferring resistance to HIV in those girls.

Latest Developments in Gene and Cell Therapies

The field of gene and cell therapies is crucial in the mainstream as drug-regulating authorities approve treatments for diseases like lymphoma and muscular dystrophy. Let us explore the latest developments regarding these therapies.

Non-Hodgkin lymphoma (NHL) accounts for about 4% of all cancers in the US, with an estimated 80,620 new cases expected this year. In this regard, Bristol Myers Squibb’s Breyanzi, a CAR T cell therapy, was approved in 2024 by the FDA, which utilizes the patient’s immune system to target and destroy cancer cells.

In 2024, the FDA approved Sarepta Therapeutics’ Elevidys, a gene therapy for Duchenne muscular dystrophy (DMD), which affects approximately 1 in 3,500 to 5000 male births worldwide, typically manifesting between ages 3 and 6. This groundbreaking offers new hope by addressing the root cause of this debilitating condition.

Exploring Current and Future Applications

CRISPR and Genome Editing: CRISPR technology has revolutionized genome editing, offering precise modifications to DNA and correcting genetic defects at their source. This technology is being explored for a variety of applications including current and future applications. However, acquiring approvals to run trials on humans has always been challenging, yet the CTX001 stands out with its success in this regard. The CTX001 is an autologous gene-edited stem cell therapy developed by CRISPR Therapeutics and Vertex Pharmaceuticals.

Dr. Haydar Frangoul, the medical director at HCA Sarah Cannon Research Institute Center, has been treating the first patient in the CTX001 trial for SCD therapy. The patient had battled sickle cell disease for 34 years before undergoing this one-time treatment. Post-treatment, her blood showed a significant proportion of fetal hemoglobin levels, enabling her to avoid blood transfusions and pain attacks without major side effects.

Stem Cell Research: These cells have the unique ability to differentiate into various cell types, making them invaluable for regenerative medicine. Research in stem cell therapy aims to treat conditions such as Parkinson’s disease, diabetes, and spinal cord injuries by replacing damaged cells with healthy ones in the near future. A notable example is a study using device-encapsulated pancreatic precursor cells derived from human embryonic stem cells. This study has shown that increased cell doses in optimized devices lead to detectable insulin production and improved glucose control.

CAR-T Cell Therapy: This therapy has shown impressive results in treating certain types of leukemia and lymphoma, offering hope for patients who have not responded to traditional treatments. This innovative approach uses modified T-cells to target and kill cancer cells. The future of CAR-T therapy looks promising, thereby expanding its application to treat more types of cancers, including solid tumors.

Gene Silencing and RNA-based Therapies: Emerging technologies like RNA interference (RNAi) and antisense oligonucleotides (ASOs) are being developed to silence harmful genes. An RNAi therapy like ‘AMVUTTRA�� developed by Alnylam, is approved in the US for treating polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. Thus, the future use of RNA therapies includes the treatment of neurodegenerative diseases like Huntington’s disease.

Understanding Ethical Considerations & the Role of Regulatory Bodies

Ethical frameworks must evolve amidst the concerns regarding ‘designer babies’, where genetic modifications used to select desired traits pose significant ethical dilemmas. A prominent example is the controversy of using CRISPR technology in human embryos, who claimed to have created the first gene-edited babies, sparking ethical debates and leading to his imprisonment. Several studies emphasize the importance of international regulatory standards and effective governance to ensure the responsible use of gene editing technologies.

Amidst the rapid pace of technological advancement, regulating gene and cell therapies needs rigorous safety standards. The regulatory bodies and agencies like the FDA’s Center for Biologics Evaluation and Research (CBER) in the US and the European Medicines Agency (EMA) in the EU play a critical role. Their frameworks include guidelines for approval of regenerative medicines and conditional or time-limited authorizations to facilitate quicker access to innovative treatments.

What the future beholds?

The future of gene and cell therapies lies in their integration into personalized medicine based on the genetic makeup of individual patients. Companies like CRISPR Therapeutics, Editas Medicine, and Intellia Therapeutics are at the forefront of research, developing therapies that could revolutionize the treatment of genetic disorders. As these therapies become more refined and accessible, they could significantly extend healthy life spans and improve the quality of life for millions.

[chuckschmalzried]

Scientist arrested for genetically editing babies

Scientist arrested for genetically editing babies https://theheartysoul.com/scientist-arrested-for-genetically-editing-babies/ Researcher He Jiankui, 35, was sentenced to three years in prison for conducting “illegal medical practices” after he announced that he had genetically edited babies. The court in Shenzhen, China, found the doctor guilty of forging approval documents from ethics review boards in order to manipulate couples, where the man had HIV and his partner […] The post Scientist arrested for genetically editing babies appeared first on The Hearty Soul. via The Hearty Soul https://theheartysoul.com/ May 17, 2024 at 09:15AM

[aneddoticamagazinestuff]

Born in China! The world's first genetically edited baby: natural resistance to AIDS

New Post has been published on https://www.aneddoticamagazine.com/born-in-china-the-worlds-first-genetically-edited-baby-natural-resistance-to-aids/

Born in China! The world's first genetically edited baby: natural resistance to AIDS

On November 26, He Jiankui, a scientist from Shenzhen, China, officially announced that a pair of genetically edited babies named Lulu and Nana were born in China in November. The twins have modified the genes to make them naturally resistant to AIDS after birth.

[iamnaturalnana]

Scientist Claims The First CRISPR Gene-Edited Babies Have Already Been Born

**Note** – if this doesn’t scare the living hell out of you, nothing will. ~ Natural Nana ~ ~~~~~~~~~~~~~~~~~~~~~~~ by DAVID NIELD A Chinese scientist is upsetting the medical world with the controversial claim he’s helped create the first gene-edited babies using CRISPR technology. Researcher He Jiankui, from the Southern University of Science and Technology in Shenzhen, China, has told the…

[vroooom2]

A brave new world revisited. It was obvious from the start that this market will turn sour.

The reality always outperforms the fiction.

That's why literature is needed: to understand the world we evolve in through great narratives. Words arranged and displayed in a beautiful manner are not only an aesthetic experience but also a breath of fresh air in the overwhelmingly formatted sentences of social media posts, newsletters, press articles... and ChatGPT.

Literature isn't the disposable marketing of storytelling. Literature isn't something you read and you forget the day after. Literature is timeless.

Hashtag: Humanities

Book to read for free, with no data stolen. Or you can just buy it in an independent bookstore, or borrow it in a public library, if you're afraid to download this pdf.

[delectablywaywardbeard-blog]

Lo scienziato cinese delle gemelline geneticamente modificate ci riprova: ora vuole curare l’Alzheimer

PECHINO – He Jiankui ci riprova. Lo scienziato cinese “padre” di tre gemelline geneticamente modificate che cinque anni fa sconvolse il mondo guadagnandosi le prime pagine di tutti i giornali, ora torna in campo con una nuova ricerca: per curare l’Alzheimer. Tutta la comunità scientifica sconfessò i suoi passati esperimenti da moderno Frankenstein: impiantò embrioni geneticamente modificati in…