Gene Therapies

Researchers at The Jackson Laboratory, Broad Institute, and Yale University use AI to identify DNA sequences that control gene activation in specific cells. This AI model decodes patterns in DNA regions called cis-regulatory elements (CREs), guiding synthetic DNA switches for cell-specific gene activity. Early testing in animal models showed succes...

A recent study from UC Davis and UC Berkeley has unveiled a groundbreaking mRNA delivery method capable of editing genes in fetal brain cells. By administering mRNA encapsulated in lipid nanoparticles, researchers successfully targeted genetic disorders such as Angelman syndrome before birth. This approach, which minimizes risks of inflammation, of...

Neurodevelopmental and psychiatric diseases (NPD), such as schizophrenia, bipolar disorder, autism, and depression, have a negative impact on individuals, families, and society. Unfortunately, effective therapies are often lacking.

He explained that he believes the human lifespan is capped at 120 years, and gene therapy might hold the key to surpassing this limit.

An 18-month-old British girl who was born completely deaf is believed to be the youngest person to have their hearing restored after undergoing groundbreaking new gene therapy.

Pfizer Inc. paused a study of an experimental gene therapy for muscular dystrophy after a child who received it died suddenly.

US regulators approved CSL Behring's hemophilia B gene therapy, a one-off infusion that frees patients from regular treatments but costs $3.5 million a dose, making it the most expensive medicine in the world.

Postnatal gene therapy may be able to prevent or repair many of the harmful effects of Pitt-Hopkins syndrome, a rare genetic condition, according to researchers at the University of North Carolina School of Medicine. In an animal model of Pitt-Hopkins syndrome, researchers discovered that restoring lost gene activity eliminates numerous clinical in...

A 10-month-old in Bengaluru, Karnataka, is fighting for her life as she waits for a medicine that costs a whopping Rs 16 crore. Diagnosed with Spinal Muscular Atrophy, type 1, the child, Khyati, needs the medicine for gene therapy before she turns 2.

Tata Memorial Hospital and IIT Bombay collaborated for the first Chimeric Antigen Receptor T-cell (CAR-T) therapy, a type of gene therapy for blood cancer treatment, in India, which was conducted on Friday, June 4 in Mumbai.

A couple from Gujarat has raised Rs 16 crore with the help of a crowdfunding platform to buy a gene therapy injection for the treatment of their 5-month-old son suffering from spinal muscular atrophy, a rare genetic disorder.

Scientists at the University of Pennsylvania and the biotech firm Regeneron are investigating whether technology developed for gene therapy can be used to make a nasal spray that will prevent infection with the new coronavirus.

A five-year-old girl with a rare neurodegenerative disease died in the US while taking part in a gene therapy trial run by French biotechnology company Lysogene, the firm said Thursday.

Two women scientists - Emmanuelle Charpentier, who is French, and American Jennifer Doudna - won the 2020 Nobel Prize in Chemistry on Wednesday for creating genetic 'scissors' that can rewrite the code of life, contributing to new cancer therapies and holding out the prospect of curing hereditary diseases.

A mouse-experiment conducted by the researchers to better understand the ways to treat renal diseases has identified that before gene therapy is implemented to treat, targeted delivery of the therapeutic genes to the kidney should be the first necessary step.

Researchers at The Jackson Laboratory, Broad Institute, and Yale University use AI to identify DNA sequences that control gene activation in specific cells. This AI model decodes patterns in DNA regions called cis-regulatory elements (CREs), guiding synthetic DNA switches for cell-specific gene activity. Early testing in animal models showed succes...

A recent study from UC Davis and UC Berkeley has unveiled a groundbreaking mRNA delivery method capable of editing genes in fetal brain cells. By administering mRNA encapsulated in lipid nanoparticles, researchers successfully targeted genetic disorders such as Angelman syndrome before birth. This approach, which minimizes risks of inflammation, of...

Neurodevelopmental and psychiatric diseases (NPD), such as schizophrenia, bipolar disorder, autism, and depression, have a negative impact on individuals, families, and society. Unfortunately, effective therapies are often lacking.

He explained that he believes the human lifespan is capped at 120 years, and gene therapy might hold the key to surpassing this limit.

An 18-month-old British girl who was born completely deaf is believed to be the youngest person to have their hearing restored after undergoing groundbreaking new gene therapy.

Pfizer Inc. paused a study of an experimental gene therapy for muscular dystrophy after a child who received it died suddenly.

US regulators approved CSL Behring's hemophilia B gene therapy, a one-off infusion that frees patients from regular treatments but costs $3.5 million a dose, making it the most expensive medicine in the world.

Postnatal gene therapy may be able to prevent or repair many of the harmful effects of Pitt-Hopkins syndrome, a rare genetic condition, according to researchers at the University of North Carolina School of Medicine. In an animal model of Pitt-Hopkins syndrome, researchers discovered that restoring lost gene activity eliminates numerous clinical in...

A 10-month-old in Bengaluru, Karnataka, is fighting for her life as she waits for a medicine that costs a whopping Rs 16 crore. Diagnosed with Spinal Muscular Atrophy, type 1, the child, Khyati, needs the medicine for gene therapy before she turns 2.

Tata Memorial Hospital and IIT Bombay collaborated for the first Chimeric Antigen Receptor T-cell (CAR-T) therapy, a type of gene therapy for blood cancer treatment, in India, which was conducted on Friday, June 4 in Mumbai.

A couple from Gujarat has raised Rs 16 crore with the help of a crowdfunding platform to buy a gene therapy injection for the treatment of their 5-month-old son suffering from spinal muscular atrophy, a rare genetic disorder.

Scientists at the University of Pennsylvania and the biotech firm Regeneron are investigating whether technology developed for gene therapy can be used to make a nasal spray that will prevent infection with the new coronavirus.

A five-year-old girl with a rare neurodegenerative disease died in the US while taking part in a gene therapy trial run by French biotechnology company Lysogene, the firm said Thursday.

Two women scientists - Emmanuelle Charpentier, who is French, and American Jennifer Doudna - won the 2020 Nobel Prize in Chemistry on Wednesday for creating genetic 'scissors' that can rewrite the code of life, contributing to new cancer therapies and holding out the prospect of curing hereditary diseases.

A mouse-experiment conducted by the researchers to better understand the ways to treat renal diseases has identified that before gene therapy is implemented to treat, targeted delivery of the therapeutic genes to the kidney should be the first necessary step.