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Broken String Biosciences and the Francis Crick Institute collaborate to advance ALS research

Partnership to expand applications of DNA break-mapping technology and advance understanding of genomic instability in development of the neurodegenerative disease amyotrophic lateral sclerosis.

From Broken String Biosciences 7 May 2024

CRISPR-based gene editing shows promise in treating rare form of blindness

About 79% of clinical trial participants experienced measurable improvement after receiving experimental, CRISPR-based gene editing that is designed to fix a rare form of blindness, according to a paper published in the New England Journal of Medicine.

6 May 2024

MedStar Washington Hospital Center enrolls first U.S. patient in gene-editing therapy trial for heart disease

The first U.S. heart patient involved in a study for a new gene-editing therapy directly infused inside the body has successfully been enrolled at MedStar Washington Hospital Center.

6 May 2024

CRISPR gene editing shows promise for treating individuals with a form of inherited blindness

Results from a groundbreaking clinical trial of CRISPR gene editing in 14 individuals with a form of inherited blindness show that the treatment is safe and led to measurable improvements in 11 of the participants treated.

6 May 2024

GenE-HumDi's strategy for involving young scientists in cutting-edge research

COST Actions are meant to be inclusive which means ensuring a geographic spread of participants, encouraging gender balance, and involving young researchers.

3 May 2024

Study highlights the role of dynamic DNA structures in memory consolidation

An international collaborative research team, including scientists from UQ's Queensland Brain Institute (QBI), has discovered a novel mechanism underlying memory involving rapid changes in a specific DNA structure.

2 May 2024

New vaccine approach shows promise in targeting less variable region of influenza virus

Duke researchers have opened a new avenue in the attack against influenza viruses by creating a vaccine that encourages the immune system to target a portion of the virus surface that is less variable.

2 May 2024

CASGEVY gene therapy eliminates vaso-occlusive crises in sickle cell patients

In a landmark study, an international consortium led by researchers at Children's Hospital of Philadelphia (CHOP) published the final results of a key clinical trial of the gene therapy CASGEVY (exagamglogene autotemcel) for the treatment of sickle cell disease in patients 12 years and older with recurrent vaso-occlusive crises (VOCs).

24 Apr 2024

Researchers receive NIH grant to help develop gene therapy for HIV

Researchers at Washington University School of Medicine in St. Louis have received a $6.2 million grant from the National Institutes of Health (NIH) to develop a gene therapy that would modify the immune system's B cells to spur them to produce broadly neutralizing antibodies against HIV.

18 Apr 2024

Targeted nanoparticle delivery of FZD4 decreases lung tumor growth and metastasis

A recent breakthrough study from the lab of Tanya Kalin, MD, PhD, professor of Child Health and Internal Medicine at the University of Arizona College of Medicine – Phoenix, has shown potential to improve therapeutic outcomes for patients suffering from lung cancers.

16 Apr 2024

Xenotransplantation poised to become a clinical reality soon

Speaking today at the Annual Meeting and Scientific Sessions of the International Society for Heart and Lung Transplantation in Prague, Muhammad Mohiuddin, MBBS, said xenotransplantation, hailed as the future of organ transplantation, is poised to become a clinical reality within the next several years.

12 Apr 2024

Gene therapy and glycoside drugs offer new hope for polycystic kidney disease treatment

Researchers have shown that dangerous cysts, which form over time in polycystic kidney disease (PKD), can be prevented by a single normal copy of a defective gene.

4 Apr 2024

Gene defect associated with argininosuccinic aciduria corrected using CRISPR-Cas9 technique

Argininosuccinate lyase deficiency (ASLD), also known as argininosuccinic aciduria, is a disease that has been enriched in the Finnish genetic heritage.

4 Apr 2024

Researchers shed light on proteins controlling the development of ovaries in mice

Researchers at the Francis Crick Institute have shed light on the proteins controlling the development of ovaries in mice before and after birth.

22 Mar 2024

Boosting mRNA activity and longevity with multi-tailed structures

Messenger RNA (mRNA) made its big leap into the public limelight during the pandemic, thanks to its cornerstone role in several COVID-19 vaccines

22 Mar 2024

Scientists shed light on key immune regulators involved in Alzheimer’s and other disorders of lipid-rich tissues

Using novel genetic and genomic tools, researchers at the Icahn School of Medicine at Mount Sinai have shed light on the role of immune cells called macrophages in lipid-rich tissues like the brain, advancing our understanding of Alzheimer's and other diseases.

21 Mar 2024

Researchers discover link between FBXW7 mutations and EGFR signaling in colorectal cancer

The Organoid group at the Hubrecht Institute have identified a new link between FBXW7 mutations and EGFR signaling activity.

18 Mar 2024

Ricoh and ERS Genomics enter into CRISPR/Cas9 license agreement

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From ERS Genomics 13 Mar 2024

New “GPS nanoparticle” delivers a genetic punch to the protein implicated in tumor spread

A newly developed "GPS nanoparticle" injected intravenously can home in on cancer cells to deliver a genetic punch to the protein implicated in tumor growth and spread, according to researchers from Penn State.

11 Mar 2024

Breakthrough in CRISPR delivery promises safer gene editing

Recent research in PNAS highlights advancements in non-viral, cell-specific delivery methods for CRISPR-Cas systems, emphasizing their potential to enhance research and gene therapy applications by minimizing risks associated with off-target effects and improving targeted delivery.

7 Mar 2024

Gene Editing News and Research

Broken String Biosciences and the Francis Crick Institute collaborate to advance ALS research

CRISPR-based gene editing shows promise in treating rare form of blindness

MedStar Washington Hospital Center enrolls first U.S. patient in gene-editing therapy trial for heart disease

CRISPR gene editing shows promise for treating individuals with a form of inherited blindness

GenE-HumDi's strategy for involving young scientists in cutting-edge research

Study highlights the role of dynamic DNA structures in memory consolidation

New vaccine approach shows promise in targeting less variable region of influenza virus

CASGEVY gene therapy eliminates vaso-occlusive crises in sickle cell patients

Researchers receive NIH grant to help develop gene therapy for HIV

Targeted nanoparticle delivery of FZD4 decreases lung tumor growth and metastasis

Xenotransplantation poised to become a clinical reality soon

Gene therapy and glycoside drugs offer new hope for polycystic kidney disease treatment

Gene defect associated with argininosuccinic aciduria corrected using CRISPR-Cas9 technique

Researchers shed light on proteins controlling the development of ovaries in mice

Boosting mRNA activity and longevity with multi-tailed structures

Scientists shed light on key immune regulators involved in Alzheimer’s and other disorders of lipid-rich tissues

Researchers discover link between FBXW7 mutations and EGFR signaling in colorectal cancer

Ricoh and ERS Genomics enter into CRISPR/Cas9 license agreement

New “GPS nanoparticle” delivers a genetic punch to the protein implicated in tumor spread

Breakthrough in CRISPR delivery promises safer gene editing

Industry focus eBook - Cell and gene therapy (2nd edition) eBook

Advancing Neuroscience Research Through Collaboration and Patient-Centered Innovation

The impact of vibration on microscopy

How Omics Technologies Are Transforming Biological Research and Drug Discovery

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