BioWorld. Link to homepage.

Clarivate
  • BioWorld
  • BioWorld Science
  • BioWorld Asia
  • Data Snapshots
    • Biopharma
    • Medical technology
    • Infographics: Dynamic digital data analysis
    • Index insights
    • NME Digest
  • Special reports
    • Infographics: Dynamic digital data analysis
    • Trump administration impacts
    • Med-tech outlook 2026
    • Under threat: mRNA vaccine research
    • BioWorld at 35
    • Biopharma M&A scorecard
    • Bioworld 2025 review
    • BioWorld MedTech 2025 review
    • BioWorld Science 2025 review
    • Women's health
    • China's GLP-1 landscape
    • PFA re-energizes afib market
    • China CAR T
    • Alzheimer's disease
    • Coronavirus
    • More reports can be found here

BioWorld. Link to homepage.

  • Sign In
  • Sign Out
  • My Account
Subscribe
BioWorld - Saturday, March 21, 2026
See today's BioWorld
Home » Prenatal gene-editing treatment offers curative potential for serious inherited disease
To read the full story, subscribe or sign in.
International Human Genome Editing Conference

Prenatal gene-editing treatment offers curative potential for serious inherited disease

March 8, 2023
By Nuala Moran
The researcher who pioneered prenatal surgery to correct neural tube defects has turned her attention to using CRISPR-edited gene therapies to correct severe monogenic diseases in utero. The availability of prenatal genetic diagnosis and advances in treating fetuses, and also in gene therapy/gene editing, make it possible to repair almost any defect in the genetic code. At the same time, there is a clear rationale for intervening before birth, Tippi MacKenzie, professor of surgery at UCSF’s School of Medicine, told attendees of the third International Human Genome Editing Conference in London on March 7.
BioWorld Science

Popular Stories

  • Today's news in brief

    BioWorld
  • Art concept for targeting the brain

    Precision psychiatry beyond, or before, biomarkers

    BioWorld
    There is broad agreement that psychiatric diagnoses in their current form are not reflective of any underlying biology, and that this is one of the things...
  • MRI image brain on black background

    ADPD 2026: Can we prevent dementia? Scientists quantify it

    BioWorld
    Neurodegenerative disease and cognitive decline cannot be explained by a single process. Beta-amyloid plaques, hyperphosphorylated tau, alpha-synuclein, activated...
  • Illustration of a motor neuron

    Keros reports beneficial effects of RKER-065 in ALS model

    BioWorld Science
    Keros Therapeutics Inc. has presented data regarding their activin receptor ligand trap, RKER-065, for the inhibition of the activin/myostatin signaling axis.
  • First-in-class POLG activator restores mtDNA across mutations

    BioWorld Science
    Researchers from Pretzel Therapeutics Inc. presented preclinical data of PX-578, a first-in-class POLG activator aimed at restoring mtDNA replication and...
  • BioWorld
    • Today's news
    • Analysis and data insight
    • Clinical
    • Data Snapshots
    • Deals and M&A
    • Financings
    • Medical technology
    • Newco news
    • Opinion
    • Regulatory
  • BioWorld Science
    • Today's news
    • Biomarkers
    • Cancer
    • Conferences
    • Endocrine/metabolic
    • Immune
    • Infection
    • Neurology/psychiatric
    • NME Digest
    • Patents
  • BioWorld Asia
    • Today's news
    • Analysis and data insight
    • Australia
    • China
    • Clinical
    • Deals and M&A
    • Financings
    • Newco news
    • Regulatory
    • Science
  • More
    • About
    • Advertise with BioWorld
    • Archives
    • Article reprints and permissions
    • Contact us
    • Cookie policy
    • Copyright notice
    • Data methodology
    • Infographics: Dynamic digital data analysis
    • Index insights
    • Podcasts
    • Privacy policy
    • Share your news with BioWorld
    • Staff
    • Terms of use
    • Topic alerts
Follow Us

Copyright ©2026. All Rights Reserved. Design, CMS, Hosting & Web Development :: ePublishing