Sickle Cell Anemia News and Research

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Sickle cell anemia is a serious disease in which the body makes sickle-shaped red blood cells. “Sickle-shaped” means that the red blood cells are shaped like a "C." Normal red blood cells are disc-shaped and look like doughnuts without holes in the center. They move easily through your blood vessels. Red blood cells contain the protein hemoglobin. This iron-rich protein gives blood its red color and carries oxygen from the lungs to the rest of the body. Sickle cells contain abnormal hemoglobin that causes the cells to have a sickle shape. Sickle-shaped cells don’t move easily through your blood vessels. They’re stiff and sticky and tend to form clumps and get stuck in the blood vessels. (Other cells also may play a role in this clumping process.) The clumps of sickle cells block blood flow in the blood vessels that lead to the limbs and organs. Blocked blood vessels can cause pain, serious infections, and organ damage.

Accelerating Genetic Research - The Story Behind One-pot DTECT

Dr Pierre Billon, PhD, grew increasingly frustrated with the lengthy turnaround times and high costs associated with obtaining genetic analysis from specialized private laboratories

8 Feb 2024

New Proof-of-Concept Model for Delivering Gene Editing Tools to Treat Blood Disorders

In a step forward in the development of genetic medicines, researchers at Children's Hospital of Philadelphia (CHOP) and the Perelman School of Medicine at the University of Pennsylvania have developed a proof-of-concept model for delivering gene editing tools to treat blood disorders, allowing for the modification of diseased blood cells directly within the body.

28 Jul 2023

Deep Learning Model Accurately Predicts RNA-Targeting CRISPR Activity

According to new research published in Nature Biotechnology, artificial intelligence can predict on- and off-target behavior of CRISPR tools that target RNA rather than DNA.

5 Jul 2023

STING-seq Discovers Causal Variants and Genetic Mechanisms for Blood Cell Traits

A major challenge in human genetics is understanding which parts of the genome drive specific traits or contribute to disease risk. This challenge is even greater for genetic variants found in the 98% of the genome that does not encode proteins.

5 May 2023

Experimental optimization of prime editing to achieve high efficiency

Genome editing technologies have enormous potential for treating genetic diseases. Techniques like the extensively used CRISPR/Cas9 gene scissors target the source of the disease directly in the DNA.

3 Feb 2023

Gene editing made faster than ever before using AI

The first simple production of customizable proteins known as zinc fingers to treat diseases by turning genes on and off might be enabled with the help of an artificial intelligence (AI) program.

From New York University (NYU) 27 Jan 2023

The complex link between pancreatic malformations and the “dark genome”

For the first time, scientists at the Centre for Genomic Regulation (CRG) have discovered a DNA sequence that is essential for pancreatic development and function.

25 Aug 2022

Researchers develop a universal AI algorithm for deep clean-up of single cell genomic data

A single cell’s genome or transcriptome can reveal considerably more data about its place in biological systems than sequencing a full batch of cells, just as interviewing a single person about their health will provide specialized, personalized information hard to obtain from a big poll.

11 Apr 2022

New gene editing approach bypasses disease-causing mutations

A new approach to gene editing using the CRISPR/Cas9 system bypasses disease-causing mutations in a gene, enabling treatment of genetic diseases linked to a single gene, such as cystic fibrosis, certain types of sickle cell anemia, and other rare diseases.

From Penn State University 22 Apr 2021