* . *
  • About
  • Advertise
  • Privacy & Policy
  • Contact
Thursday, June 5, 2025
Earth-News
  • Home
  • Business
  • Entertainment
    ‘Sinners,’ starring Michael B. Jordan, is now streaming on Prime Video – About Amazon

    Experience the Thrills of ‘Sinners’ Starring Michael B. Jordan – Now Streaming on Prime Video!

    California Mid-State Fair announces entertainment lineups for Frontier and Mission stages – KSBY News

    Exciting Entertainment Lineup Unveiled for California Mid-State Fair’s Frontier and Mission Stages!

    Spotify & OpenAI: Will Gen AI ‘Hollow Out’ Entertainment? – Technology Magazine

    Will Generative AI Transform the Future of Entertainment

    Author Richard Russo Will Speak At Sandwich Town Hall – CapeNews.net

    Join Us for an Inspiring Evening with Author Richard Russo at Sandwich Town Hall!

    Entertainment Partners Acquires CASHét, Digital Payments Vendor for Productions – Variety

    Entertainment Partners Acquires CASHét, Digital Payments Vendor for Productions – Variety

    Salem’s Harborwalk Garden Cultivates Community with Entertainment, Food, and Events – 105.7 WROR

    Discover the Vibrant Community Spirit at Salem’s Harborwalk Garden: A Hub for Entertainment, Food, and Fun!

  • General
  • Health
  • News

    Cracking the Code: Why China’s Economic Challenges Aren’t Shaking Markets, Unlike America’s” – Bloomberg

    Trump’s Narrow Window to Spread the Truth About Harris

    Trump’s Narrow Window to Spread the Truth About Harris

    Israel-Gaza war live updates: Hamas leader Ismail Haniyeh assassinated in Iran, group says

    Israel-Gaza war live updates: Hamas leader Ismail Haniyeh assassinated in Iran, group says

    PAP Boss to Niger Delta Youths, Stay Away from the Protest

    PAP Boss to Niger Delta Youths, Stay Away from the Protest

    Court Restricts Protests In Lagos To Freedom, Peace Park

    Court Restricts Protests In Lagos To Freedom, Peace Park

    Fans React to Jazz Jennings’ Inspiring Weight Loss Journey

    Fans React to Jazz Jennings’ Inspiring Weight Loss Journey

    Trending Tags

    • Trump Inauguration
    • United Stated
    • White House
    • Market Stories
    • Election Results
  • Science
  • Sports
  • Technology
    Domo to Participate in the D.A. Davidson Technology Summit – Business Wire

    Domo Set to Shine at the D.A. Davidson Technology Summit!

    When fiction becomes fact: 3 pieces of modern technology inspired by Star Trek – Redshirts Always Die

    From Screen to Reality: 3 Modern Technologies Inspired by Star Trek

    The Isfahan Center for Nuclear Technology – UCF, ZPP, and IRR10 – Alma Research and Education Center

    Exploring the Isfahan Center for Nuclear Technology: Innovations and Insights from UCF, ZPP, and IRR10

    Inside the tedious effort to tally AI’s energy appetite – MIT Technology Review

    Inside the tedious effort to tally AI’s energy appetite – MIT Technology Review

    Finland Set to Lead EU Quantum Technology Defense Project – IoT World Today

    Finland Set to Lead EU Quantum Technology Defense Project – IoT World Today

    AI for lawyers: Win back your time using technology – nationaljurist.com

    Reclaim Your Time: How AI is Transforming the Legal Profession

    Trending Tags

    • Nintendo Switch
    • CES 2017
    • Playstation 4 Pro
    • Mark Zuckerberg
No Result
View All Result
  • Home
  • Business
  • Entertainment
    ‘Sinners,’ starring Michael B. Jordan, is now streaming on Prime Video – About Amazon

    Experience the Thrills of ‘Sinners’ Starring Michael B. Jordan – Now Streaming on Prime Video!

    California Mid-State Fair announces entertainment lineups for Frontier and Mission stages – KSBY News

    Exciting Entertainment Lineup Unveiled for California Mid-State Fair’s Frontier and Mission Stages!

    Spotify & OpenAI: Will Gen AI ‘Hollow Out’ Entertainment? – Technology Magazine

    Will Generative AI Transform the Future of Entertainment

    Author Richard Russo Will Speak At Sandwich Town Hall – CapeNews.net

    Join Us for an Inspiring Evening with Author Richard Russo at Sandwich Town Hall!

    Entertainment Partners Acquires CASHét, Digital Payments Vendor for Productions – Variety

    Entertainment Partners Acquires CASHét, Digital Payments Vendor for Productions – Variety

    Salem’s Harborwalk Garden Cultivates Community with Entertainment, Food, and Events – 105.7 WROR

    Discover the Vibrant Community Spirit at Salem’s Harborwalk Garden: A Hub for Entertainment, Food, and Fun!

  • General
  • Health
  • News

    Cracking the Code: Why China’s Economic Challenges Aren’t Shaking Markets, Unlike America’s” – Bloomberg

    Trump’s Narrow Window to Spread the Truth About Harris

    Trump’s Narrow Window to Spread the Truth About Harris

    Israel-Gaza war live updates: Hamas leader Ismail Haniyeh assassinated in Iran, group says

    Israel-Gaza war live updates: Hamas leader Ismail Haniyeh assassinated in Iran, group says

    PAP Boss to Niger Delta Youths, Stay Away from the Protest

    PAP Boss to Niger Delta Youths, Stay Away from the Protest

    Court Restricts Protests In Lagos To Freedom, Peace Park

    Court Restricts Protests In Lagos To Freedom, Peace Park

    Fans React to Jazz Jennings’ Inspiring Weight Loss Journey

    Fans React to Jazz Jennings’ Inspiring Weight Loss Journey

    Trending Tags

    • Trump Inauguration
    • United Stated
    • White House
    • Market Stories
    • Election Results
  • Science
  • Sports
  • Technology
    Domo to Participate in the D.A. Davidson Technology Summit – Business Wire

    Domo Set to Shine at the D.A. Davidson Technology Summit!

    When fiction becomes fact: 3 pieces of modern technology inspired by Star Trek – Redshirts Always Die

    From Screen to Reality: 3 Modern Technologies Inspired by Star Trek

    The Isfahan Center for Nuclear Technology – UCF, ZPP, and IRR10 – Alma Research and Education Center

    Exploring the Isfahan Center for Nuclear Technology: Innovations and Insights from UCF, ZPP, and IRR10

    Inside the tedious effort to tally AI’s energy appetite – MIT Technology Review

    Inside the tedious effort to tally AI’s energy appetite – MIT Technology Review

    Finland Set to Lead EU Quantum Technology Defense Project – IoT World Today

    Finland Set to Lead EU Quantum Technology Defense Project – IoT World Today

    AI for lawyers: Win back your time using technology – nationaljurist.com

    Reclaim Your Time: How AI is Transforming the Legal Profession

    Trending Tags

    • Nintendo Switch
    • CES 2017
    • Playstation 4 Pro
    • Mark Zuckerberg
No Result
View All Result
Earth-News
No Result
View All Result
Home Health

3D genome architecture influences SCID-X1 gene therapy success

October 6, 2023
in Health
3D genome architecture influences SCID-X1 gene therapy success
Share on FacebookShare on Twitter

3D genome architecture influences SCID-X1 gene therapy success

Genomic signatures of lentiviral VIS. (A) Circular projection of the human genome with integration sites from patients 1, 2, 3, and 6. Gene names near integration site hotspots are listed in the inner circle. (B) Overlap of VIS hotspots in patients. The numbers along the diagonal represent the number of hotspots in individual patients, and the other numbers indicate hotspots common between pairs of patients. (C) RIGs in patients 1, 2, 3, and 6. Word clouds show the frequency of integration site clustering at each of the genes. Genes with more VISs within them are shown in larger font. Patients 2, 3, and 6 share similar RIGs. (D) Top RIGs among patients. Many RIGs were shared by patients 2 through 10; patient 1 had a set of unique RIGs. (E) The proportion of VISs located in various genomic regions. Most VISs fall in introns. (F) Enrichment of VISs in various genomic regions. The enrichment is calculated by normalizing the number of VIS fall upon a region by a background, in which is the number of VIS merely scales with the length of the region. Credit: Science Advances (2023). DOI: 10.1126/sciadv.adg9959

Patients with X-linked severe combined immunodeficiency disorder (SCID-X1), sometimes called “bubble boy disease,” are born with a defective gene that prevents them from producing immune cells. Gene therapy from St. Jude Children’s Research Hospital restored the immune system in multiple infants with SCID-X1 in 2019 by supplying copies of the corrected gene.

Through ongoing efforts to monitor patient safety, St. Jude scientists recently documented where the gene copies integrate into patient DNA, providing a foundation to understand the biology and safety of using lentiviral vectors. The findings were published today in Science Advances.

“We now have a robust pipeline to monitor the safety of lentiviral gene therapies,” said senior co-corresponding author Jiyang Yu, Ph.D., St. Jude Department of Computational Biology interim chair. “This really gives hope to patients with genetic diseases that can be cured by lentiviral gene therapy. It looks like we cured bubble boy disease safely in these patients.” The St. Jude team published the entire computational pipeline together with the manuscript.

Several years after treatment, the St. Jude lentiviral gene therapy for SCID-X1 appears both effective and safe, unlike earlier retroviral approaches. The researchers identified where the gene was added to patients’ DNA and why the vector integrated in that particular place. Scientists previously knew that lentiviral gene therapies integrated into different areas of DNA that appeared safer than earlier technologies, but they could not address why.

3D genome structure holds the key to SCID-X1 gene therapy

The scientists found that copies of the new, corrected gene had been inserted into certain genomic hotspots for the patients in the study. The reason was deceptively simple: From a 3D structural perspective, the hotspots are regions the lentiviral vector first encounters after entering the cell’s nucleus through a channel called a nuclear pore.

“It’s like someone coming into a room and taking the first available seat near the door,” said co-corresponding Stephen Gottschalk, M.D., St. Jude Department of Bone Marrow Transplantation and Cellular Therapy chair. “The room is the nucleus. The seats are these DNA elements right near the door of the nuclear pore. That never occurred to me before this study, but it’s a very simple principle in the end.”

The St. Jude group found that the integration site pattern of the gene therapy into patient cells shed light on the safety and efficacy of the approach.

“We performed a comprehensive single-cell multi-omic analysis of this gene therapy to understand whether a functional copy of the corrected gene was in patient cells, to what extent the gene was expressed, and the chromatin organization at a single-cell level,” said first author Koon-Kiu Yan, Ph.D., St. Jude Department of Computational Biology. “Before this study, we could measure the general gene expression of a bulk group of cells. But with bone marrow samples from two patients, we saw at a single-cell level which genes were expressed in which cell types.”

Using that single-cell analysis, along with their other work, Yan showed that the safety and efficacy of treatment are also related to integration into compartments near the nuclear pore. Previous gene therapy efforts integrated into promoters near to or directly into oncogenes, ultimately causing cancer.

The lentiviral vector used by St. Jude selectively avoids promoters without disrupting oncogenes, improving treatment safety. The same pattern was observed with a lentiviral gene therapy used to create chimeric antigen receptor (CAR) T cells, suggesting that the phenomenon may be a general mechanism not restricted to the SCID-X1 vector.

“The integration pattern data could serve as a map of potentially safe integration sites,” Yu and Gottschalk explained. “The single-cell analysis is like deep cartography, a map with a near pixel-perfect resolution. The large number of integration sites could be used as a safety reference for future lentiviral gene therapies.”

One case was especially notable: the patient who required a second dose of the gene therapy to achieve a cure was found to have a different integration pattern than the patients who had responded to the first dose. After receiving the second dose, the patient’s integration pattern resembled the others,” and the therapy was effective. This study provides a foundation for understanding differences in treatment response so that future gene therapies may be improved.

More information:
Koon-Kiu Yan et al, Integrome signatures of lentiviral gene therapy for SCID-X1 patients, Science Advances (2023). DOI: 10.1126/sciadv.adg9959. www.science.org/doi/10.1126/sciadv.adg9959

Citation:
3D genome architecture influences SCID-X1 gene therapy success (2023, October 6)
retrieved 6 October 2023
from https://medicalxpress.com/news/2023-10-3d-genome-architecture-scid-x1-gene.html

This document is subject to copyright. Apart from any fair dealing for the purpose of private study or research, no
part may be reproduced without the written permission. The content is provided for information purposes only.

>>> Read full article>>>
Copyright for syndicated content belongs to the linked Source : Medical Xpress – https://medicalxpress.com/news/2023-10-3d-genome-architecture-scid-x1-gene.html

Tags: ArchitectureGenomehealth
Previous Post

Study discovers possible tool to diagnose common contributor to vascular dementia

Next Post

What I’ve Learned from my Patients about the Challenges of Inoperable Lung Cancer

The Ecology of Visitation – National Park Service (.gov)

Exploring the Impact of Visitor Interactions on Natural Ecosystems

June 5, 2025
Students play with science at Camp Invention at School of Innovation – News-Herald

Unleashing Creativity: Students Dive into Science at Camp Invention!

June 5, 2025
Are cold plunges good for you? Here’s what the science says. – The Washington Post

Unlocking the Benefits of Cold Plunges: What Science Reveals!

June 5, 2025
Casino magnate Alvin Chau’s wife Heidi Chan embraces Buddhist lifestyle after husband’s incarceration – VnExpress International

Casino magnate Alvin Chau’s wife Heidi Chan embraces Buddhist lifestyle after husband’s incarceration – VnExpress International

June 5, 2025
How each of the 32 teams qualified for the 2025 Club World Cup – The New York Times

Unveiling the Path: How All 32 Teams Earned Their Spot in the 2025 Club World Cup

June 5, 2025
Service side of the U.S. economy contracts for first time in almost a year due to trade fights – MarketWatch

U.S. Service Sector Shrinks for the First Time in Nearly a Year Amid Trade Tensions

June 5, 2025
‘Sinners,’ starring Michael B. Jordan, is now streaming on Prime Video – About Amazon

Experience the Thrills of ‘Sinners’ Starring Michael B. Jordan – Now Streaming on Prime Video!

June 5, 2025
Sutter Health investing $23 million in primary and behavioral care – Healthcare Finance News

Sutter Health investing $23 million in primary and behavioral care – Healthcare Finance News

June 5, 2025
Gov. Shapiro sues USDA over canceled $13M food purchasing program – LancasterOnline

Gov. Shapiro sues USDA over canceled $13M food purchasing program – LancasterOnline

June 5, 2025
Domo to Participate in the D.A. Davidson Technology Summit – Business Wire

Domo Set to Shine at the D.A. Davidson Technology Summit!

June 5, 2025

Categories

Archives

June 2025
MTWTFSS
 1
2345678
9101112131415
16171819202122
23242526272829
30 
« May    
Earth-News.info

The Earth News is an independent English-language daily published Website from all around the World News

Browse by Category

  • Business (20,132)
  • Ecology (667)
  • Economy (681)
  • Entertainment (21,587)
  • General (15,261)
  • Health (9,723)
  • Lifestyle (684)
  • News (22,149)
  • People (682)
  • Politics (690)
  • Science (15,901)
  • Sports (21,185)
  • Technology (15,667)
  • World (668)

Recent News

The Ecology of Visitation – National Park Service (.gov)

Exploring the Impact of Visitor Interactions on Natural Ecosystems

June 5, 2025
Students play with science at Camp Invention at School of Innovation – News-Herald

Unleashing Creativity: Students Dive into Science at Camp Invention!

June 5, 2025
  • About
  • Advertise
  • Privacy & Policy
  • Contact

© 2023 earth-news.info

No Result
View All Result

© 2023 earth-news.info

No Result
View All Result

© 2023 earth-news.info

Go to mobile version