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Gene and Cell Therapy – News and Features

Above image shows a metastatic tumor in the left lung of a patient and the bottom image shows no tumor following treatment.

Immunotherapy Approach May Be Effective for Some Metastatic Tumors

Early findings from a small clinical trial provide evidence that a new cellular immunotherapy approach may effectively treat metastatic solid tumors.
 A DNA double helix being "edited" with a pair of tweezers.

Novel Genome Editing Approach Restores Hearing in Mouse Model of Inherited Deafness

Researchers have successfully restored hearing in a mouse model of inherited deafness using a novel in vivo gene editing approach.
Hospital beds on a ward.

Cell-Based Lymphoma Therapy Shows Promise in Trial

CAR-T cell therapy causes tumors to shrink or disappear, but if this CAR-T treatment fails, the median survival time after relapse is about six months. Now, a phase 1 clinical trial has found that a new CAR-T cell therapy significantly improved these patients’ outcomes.
Immune cells attacking a cancer cell.

Researchers Create Novel Cell Type That Kills Cancer Cells in Research Models

Researchers have combined the genetic components of four types of cells to make a powerful new cell type, Co-STAR (Co-stimulatory Synthetic T-cell receptor and Antigen Receptor) cells. These cells were able to produce a sustained anti-tumor response against human cancer cells in laboratory studies.
Two cytotoxic T cells attack a cancer cell.

Stem Cell-Derived Therapy Shows Early Promise for Resistant Liver Cancer

An innovative new stem cell-derived therapy could be used to better target and treat the most common form of liver cancer.
A gloved hand holds a syringe filled with yellow liquid.

World-First CAR T-Cell Therapy for Child With Lupus

CAR T-cell therapy has been used for the first time to treat lupus in a child, restoring the patient's ability to live a normal life.
Magnifying glass placed over a jigsaw piece with the word "quality" written on it.

Create Therapies Confidently With Quality by Design

In this article, one of the leading quality control strategies being implemented by pharmaceutical manufacturers is discussed.
A stem cell dividing in two with other cells and DNA helices in the background

Unmasking the True Identity of Gut Stem Cells

Columbia researchers have found that Lgr5+ cells descend from the gut’s true stem cells. This case of mistaken identity may explain why regenerative medicine has not lived up to its promise.
Cancer cells.

Study Sheds Light on Early Development of Multiple Myeloma

Multiple myeloma is one of the most common forms of cancer of the immune cells in the bone marrow. It is considered incurable. Even when patients respond to treatment at first, the cancer comes back.
A white and blue strand of DNA.

Gene Therapy Halts Progression of Young Boy’s Rare Genetic Condition

Gene therapy has slowed the progression of an "ultra-rare" progressive neurodegenerative disorder.